Page 11234..»

Archive for the ‘Gene Therapy Doctor’ Category

Selwa Al-Hazzaa: The Saudi doctor giving the gift of sight – Arabnews

RIYADH: Professor Selwa Al-Hazzaa is a Saudi female success story set on the road to excellence from childhood.

Speaking to Arab News, Al-Hazzaa, an ophthalmologist and chairman of the ophthalmology department at King Faisal Specialist Hospital and Research Center (KFSHRC), told of her 27 years of devoted work to bettering the health care system, becoming the first woman to hold a high position at the hospital where she dedicated her life, energy and time to making a difference in her field.Al-Hazzaas career took off in 1995, as the first Saudi woman to be made a member of the Medical Advisory Council at King Faisal Hospital. Her journey wasnt the easiest, but with her talent, hard work and ambition, she was recognized by the Saudi leadership early on and used the platform to pave the way for future women in medicine and other fields.Born into a family of five girls, she grew up in Tucson, Arizona in the 1960s while her father was completing his studies. She excelled in her school years, always living up to the highest standards and expectations which she has placed upon herself.

I didnt choose ophthalmology, ophthalmology chose me.

Prof. Selwa Al-Hazzaa

I went into medicine not wanting (to do) it, she said. Nevertheless, she put all her energy into studying, because she had a higher ambition and was keen to make a difference.One of her biggest challenges was when it was time for her to enroll in university. She wanted to travel abroad to study, but was unable to, because it was rare for women to do so at the time.Back then the only two real professional options women had were medicine or education, and her father gave her a choice: Either to become a teacher or a physician. She chose the latter.After obtaining her medical degree from King Saud University, she did her fellowship at the Wilmer Ophthalmologic Institute at Johns Hopkins University School of Medicine, in Washington, DC.

HIGHLIGHTS

Selwa Al-Hazzaa became the first woman to hold a high position at the hospital where she dedicated her life, energy and time to making a difference in her field.

With her talent, hard work and ambition, she was recognized by the Saudi leadership early on and used the platform to pave the way for future women in medicine and other fields.

Her first patient was a 9-year-old Saudi girl born blind, a case Al-Hazzaa had followed since the girl was less than a year old.

She returned to the Kingdom, where she was later chosen by the head of KFSHRC, Dr. Anwar Jabarti, to be the late King Fahds ophthalmologist. She credits Jabarti for realizing her potential, dedication and skills by looking beyond gender and solely at talent.Her dream of representing her country came true, though under sombre circumstances, when she went on her first diplomatic mission after the fatal Sept. 11 2001 terror attacks in the US, remembering her fathers words: When people trust you, they will then let you represent the country.

Selwa Al-Hazzaa. (AN photo by Ali Aldhahri)

And represent her country she did, as she was the only woman between men, and with no training whatsoever in the political arena, she spoke from the heart, connecting with people. From that day on, the government took me as their voice of Saudi Arabia after Sept. 11.Through a lifetime of giving, people would ask her what was the secret to her success. There is no secret females are always givers. When we are young, we take care of our siblings, when we are married we take care of our husbands, we get pregnant and take care of our children, she said.Elected as an executive member of the International Council of Ophthalmology (ICO) in 2002, she became the youngest member, the first woman member from the Middle East, and the only female on the council from 2002-2006. She stood down in 2010.

NUMBER

2.2bn - There are an estimated 2.2 billion people with vision impairment or blindness globally, with an estimated 1 billion who suffer from moderate or severe distance vision impairment or blindness (WHO 2019).

In 2017, Al-Hazzaa was granted the degree of doctor of humanities, honoris causa, the highest honor at Franklin University, one of many honorary titles shes received in her career. She is also a member of various editorial boards, fellowships and committees, and was one of the first group of women appointed to the Saudi Shoura Council by late King Abdullah bin Abdul Aziz in 2003, in a historic move, allowing women for the first time to be part of the Kingdoms formal advisory body.

FASTFACT

Last November, Selwa Al-Hazzaa, alongside her colleague Dr. Mohamed Khuthaila and a medical team consisting of entirely of Saudis, put the Kingdom on the map as the first country in the Middle East, and the 5th globally, to utilize LUXTURNA, the first USA FDA-approved gene therapy treatment for any genetic disorder to treat blindness in children.

With her 27 years of experience in the field, publishing 69 accredited papers and more, her lifes work finally paid off in November of last year when she, alongside her colleague Dr. Mohamed Khuthaila and a medical team consisting entirely of Saudis, put the Kingdom on the map as the first country in the Middle East, and the 5th globally, to utilize LUXTURNA, the first USA FDA-approved gene therapy treatment for any genetic disorder to treat blindness in children.Her first patient was a nine-year-old Saudi girl born blind, a case Al-Hazzaa had followed since the girl was less than a year old. The successful utilization of the treatment was one of her finest career achievements to date.If you are going to take a certain specialty, dont take what everybodys taking take something that doesnt exist and make it exist. Take something hard, because then when you are called upon, it will be regardless of your gender.I didnt choose ophthalmology, ophthalmology chose me.

Read more:
Selwa Al-Hazzaa: The Saudi doctor giving the gift of sight - Arabnews

A baby who can’t crawl: A cure is on the way, but this Milton baby’s time is running out – InsideHalton.com

"We're moving close to a cure," says Wyatt's doctor, Mark Tarnopolsky, who is director of the neuromuscular and neurometabolic clinic at McMaster University Medical Centre in Hamilton.

Children with spinal muscular atrophy lack a certain protein that is critical for the maintenance and function of specialized nerve cells, called motor neurons, which control muscle movement throughout the body.

Without the protein, the motor neurons die and the muscles can't move. If they don't move, they shrink and weaken. The child suffers debilitating effects that inhibit even their ability to breathe and swallow.

Until recently, most children with this type of spinal muscular atrophy died of respiratory failure by their second birthday.

But in 2018, a new drug called Spinraza was approved for use in Canada. It prevents the rapid nerve degeneration from occurring, if it is given early enough in the child's life.

At $125,000 a dose, it's very expensive. Luckily, the Ontario government pays for it.

Wyatt has had four doses since he was diagnosed six months ago.

He can clap his hands together and kick a little bit, Dannon said.

"He is such a happy little guy," she said. "Every moment I spend with him, I love."

Wyatt's aunt and parents have started a GoFundMe page to help pay for some of his expenses. The family has already raised about $74,000 through this and other fundraisers.

They've poured their hopes into a brand new drug called Zolgensma. It's a gene therapy does the same thing as Spinraza, but it's a single dose that lasts 25 years.

"With a longing we didn't know was possible, we hope one day Wyatt can dance to the beat of his favourite song, send our hearts racing as he climbs a high tree or wrestle with his dad on the floor," the family says on its GoFundMe page.

But that's unlikely to happen.

The drug is very expensive, at $2.8 million Canadian for a dose. It's not available in Canada yet.

More significantly, Tarnopolsky thinks the benefits would be very limited for a child like Wyatt. That's because he's already too old.

Nerve degeneration happens very quickly for the tiny number of children, perhaps several hundred in Ontario, who have this disease. It drops "like a stone" shortly after birth, Tarnolpolsky said.

And once the nerve function is gone, it doesn't come back.

The studies have shown it's much better for children to receive either Spinraza or Zolgensma if they get it when they're two or three months old. The oldest child in one of the Zolgensma studies was eight months old.

He would like to see newborns routinely screened for this disease so it can be treated right away.

Michael Harris, the Waterloo regional councillor who was formerly MPP for Kitchener-Conestoga riding, has met many families like the Vaseys.

"They'll do anything" for a chance for their child to get better," he said.

When he was MPP between 2011 and 2018, Harris championed the cause of families with rare diseases.

He toured the province to hear from them, tried to have an all-party committee of MPPs to discuss the issues, and lobbied for them to have access to government funds for the expensive medication they need. (It's expensive because so few patients need it, and there are few buyers to share the high cost of research, development and clinical trials.)

Working for these families was "the only time I came to tears in my entire career," he said. "I think of them all the time."

One look at Wyatt helps explain why.

He is cheerful all the time despite the long uphill road ahead for him, his family said.

Now that he has learned to clap his hands together, "all he loves to do now is clap," said Dannon.

"You would never know any of the challenges (that he faces) exist for him."

ldamato@therecord.com

Twitter: @DamatoRecord

ldamato@therecord.com

Twitter: @DamatoRecord

See the original post here:
A baby who can't crawl: A cure is on the way, but this Milton baby's time is running out - InsideHalton.com

UCLA’s Fight to Patent a Life-Saving Cancer Drug Could Make the Medicine Virtually Unobtainable in India – LA Magazine

Note: This article was co-published with the Daily Bruinas a product of the Bridget O Brien travel grant.

Sarat Kumar Borah had been taking enzalutamide for six weeks when he told his son he wanted to give up. The medicine was helping a little with his prostate cancer symptomshis headaches were milder, some days he had more energybut he became livid whenever he thought about the price of the drug.Im drying up your funds, he told his son. I am old. What is the point of me living any longer if its going to finish everyones reserves.

Without insurance to cover the cost of the medication, Borah had relied on his son, Rupam, to help pay for the drugs. Even with the leftover box of the medicine Sarats oncologist gave him, Rupam estimates he spent around 25 thousand rupees on treatments, nearly a fourth of the average annual salary in India.

Rupam insists his father is not the type to give up.

Im going to fight this damn thing out, his father had joked just weeks earlier. Who do you think youre dealing with?

But things had changed.

Although he owns a successful design business, Rupam stopped eating out, going on vacations, and dipped far into his youngest daughters college fund, leaving him up most nights trying to piece together a plan to send her to university.

Im just trying to tell her, Finish your class 12 and hold on, lets see how we do, he says. I cant break her heart.

Even if the medicine was just a little less expensive it would help, he said. Instead, in the next couple months, the price of enzalutamide could dramatically increase.

In 2016, the patent for enzalutamide, a wonder drug for late-stage prostate cancer discovered by UCLA, was rejected by the Indian patent office, a decision many believed would make the drug less expensive.

Generic versions of the drug flooded the market, offering enzalutamide for a fraction of the cost of the name-brand drug, Xtandi. Although it still wasnt as cheap as chemotherapy, Knowledge Ecology International, a consumer advocacy group, estimated that as more generic companies joined the market, the cost of a pill could be driven down to 50 cents.

Then, just a year later, UCLA appealed the decision to the Delhi High Court and, last May, won. The court sent the patent back to the Indian patent office to evaluate anew. If the office approves the patent, the decision would outlaw the generic brands of enzalutamide, leaving only Xtandi, a medication with a daily dose that is 70 percent more expensive, costing about 2 lakhs for 112 capsules, or about $223,576 in U.S. dollars.

Liz Ketcham

In 2007, the UC system and 11 other universities signed on to a set of ethical licensing guidelines that emphasized the consideration of the needs of people in developing countries, according to the document. But to critics, the pledge was little more than a PR stunt. As it was signing its symbolic goodwill gesture, UCLA was also wrapping up the licensing agreement for Xtandi, with no provisions to ensure patients in developing countries would be able to afford the life-saving medication.

Phil Hampton, a UCLA spokesperson, says the university has since changed its licensing policies to include language that encourages licensees to consider the interests of underserved populations.

Still, Rupam Borah and countless others will not be able to afford the medication if the patent is approved. Spending a quarter of his salary on medicine is unaffordable spending 40 times his salary is impossible.

We will have to live by the reality that he will have to pass away sooner, he says. Thats it. What else can we do?

In a small lab on the southern end of UCLAs campus, Michael Jung spent most of his career as an organic chemist, developing new ways to synthesize chemicals. For 45 years he was content tinkering away at basic research questions and publishing results in journals like Tetrahedron Letters. Then on his 55th birthday, his wife pulled him aside.

What do you want to do for the rest of your life, she asked. More of the same?

Jung said he always dreamed of finding a cure for a human disease but never had the guts to try it. Some researchers worked years on developing a medication with nothing to show for it.

The day after his birthday, a colleague asked if he wanted to join a project dedicated to creating a prostate cancer drug. He took it as a sign, even though he knew almost nothing about prostate cancer. If nothing came of it, the worst people would think was that hed decided to retire early, he thought.

A couple years later, in 2009, he published a paper in Science, introducing enzalutamide, a molecule that could drastically alter the way prostate cancer was treated. While chemotherapy moves through the body quickly and indiscriminately, wiping out cancer cells and any other cells that divide quickly (like bone marrow), enzalutamide elegantly clicks into receptors on the surface of prostate cancer cells, blocking the hormones that cause them to grow.

Stephen Freedland, an oncologist at Cedars-Sinai, remembers the excitement around enzalutamide and other similar drugs.

There werent a lot of options for men. It was kind of like, Sorry, life sucks, Freedland says. Then all of a sudden we had a drug that could salvage a lot of these guys and just buy them time, a good quality of life.

Chemotherapy often leaves patients bed ridden with nausea. In contrast, patients on enzalutamide can usually maintain their normal lifestyles, only complaining occasionally about feeling a bit foggy. One of Freedlands patients said he couldnt lift as much at the gym, which is to say he, a late-stage cancer patient, was still working out.

In early clinical trials, late-stage prostate cancer patients who had already exhausted hormone therapies and chemotherapy, enzalutamide extended their lives by five months. In oncologist circles, the result was promisingusually treatments that work in late-stage cancer patients work even better for early stage patients.

Two years later, scientists stopped a clinical trial short after enzalutamide significantly reduced the risk of death in patients before they started chemotherapy, and in August, the FDA approved enzalutamide for patients before they even started hormone therapy.

Freedland thinks eventually enzalutamide could turn prostate cancer into a chronic illness something you can live with for years like diabetes or asthma.

We are in a renaissance period in the medical therapy of prostate cancer, an author of one of the clinical trials for enzalutamide said. Even at this early stage, enzalutamide is a game changer.

By weight, Xtandi is 65 times more expensive than gold. In the United States, that is 2.5 times the annual per capita income. In India, the name-brand drug costs more than 38 times the per capita income.

Rajeev Kumar, a urologist at a public hospital in Delhi, says if the patent were approved, almost none of his patients could afford the medication. Already, his patients struggle to afford the generic brands. Many of them run out of money a couple weeks into the treatment plan and stop taking the pills. Others, aware theyll inevitably exhaust their resources, refuse the prescription from the beginning.

With the patent, even his wealthiest patients would be unable to afford the medicine.

Liz Ketcham

In the United States, 80 percent of patients with insurance have a copay of less than $25 a month for Xtandi. In India, Kumar estimates less than 20 percent of his patients have insurance.If they do, their insurance policies usually cover a fixed amount of medical expenses for the entire family, which Xtandi would quickly deplete.

Rupam Borah has seen the worst of it in the hospital as he waits for his fathers appointmentschildren, mothers, and wives sobbing next to him, coming to terms with the fact that they cant afford medication.

Im still pushing for medicines and going desperate and having sleepless nights sometimes and all that but still look at people who are below us who cannot even imagine they will die, he says, shaking his head. Ive seen those kinds of people in hospitals. Ive seen them. I mean, they just cry. They are completely helpless.

Most of the men hes talked to in the hospital can scrape together enough for about a fourth of the cost of one box of enzalutamide. Sometimes he gives money to other patients at the hospital. Stretch it for as long as you can, he tells them.

If the pills became more expensive, Kumar isnt sure he would even prescribe enzalutamide to his patients anymore. A couple of months of life is only worth so much financial stress.

I know if I offered it to him he would take it and thatll end up destroying his family, he says. The next generation (will be) in debt for the next 10 years.

Even as an established doctor and professor at the most widely respected hospital in Delhi, one box of Xtandi would cost his entire monthly salary.

Would I give it all up to extend a couple of months? he says. Probably not.

Enzalutamide ultimately made the University of California more than a billion dollars. Even for the UC system, which owns the most patents of any American university, the drug generated more money than any other patent sale.

We are strategically supporting one of our essential missionsfunding and generating research with practical applications that serve the public good , Gene Block, the chancellor of UCLA, said in a university press release following the deal.

Except to many, it didnt seem as if the university was trying to serve the public good.

First, there was the billion dollars.

To R. Joseph Trojan, a pharmaceutical patent lawyer based in Los Angeles, it was simple economics: the more the UC charged for the patent, the higher pharmaceutical companies would have to price the medicine to recover what they had spent.

Where does it think the money was going to come from? he asks. It was coming out of the pockets of people who need the drug. Why are you driving up the cost of these drugs by demanding a billion dollar licensing fee upfront? That makes no sense at all.

Furthermore, although researchers used public funding from the National Institutes of Health and the U.S. Armys prostate cancer research program to discover Xtandi, UCLA was actively pursuing a patent that would make it inaccessible to the public. Meanwhile, money from the patent sale sits in a portfolio, generating $60 million dollars for UCLA every year.

Then there was the language in the licensing agreement. With no clause to protect developing countries, the agreement seemed to directly contradict the universitys own licensing guidelines, which say it should consider such public benefit and broad societal needs when developing licensing strategies.

John Mazziotta, the CEO of UCLA Health, says these ethical licensing guidelines are not always prescriptive, explaining there are a variety of complex and potentially contradictory issues that need to be taken into account when drafting such an agreement. Producing and testing medicine is expensive, Mazziotta argues. A pharmaceutical company might not agree to produce the medicine at all if there were a clause limiting the sales in developing countries.

Healthcare advocates have long been skeptical of that argument given that other companies, like the French NGO responsible for a successful gene therapy, included a reasonable pricing clause in their agreement and were still able to license the patent for millions.

Other institutions, like Harvard University, were able to include global access provisions in more than half of their pharmaceutical licensing agreements, clauses that allow generic companies in developing countries to produce their patented medications. They also allow Harvard to refuse to prosecute a patent in a developing country.

The UC created a committee in 2009 to oversee the inclusion of clauses that consider the needs of underserved people. These clauses have been included in 30 licensing agreements since 2018. Still, some argue this change was too little too late.

Without a global access provisions in the Xtandi agreement, if Medivation, a biopharmaceutical company that helped manufacture Xtandi, wanted UC to prosecute a patent that could take away medicine from thousands, the university has no legal avenue to refuse.

UC ultimately appealed the India patent decision on a technicality, claiming the patent office hadnt taken into account a piece of evidence. To argue its case, they hired Palaniappan Chidambaram, the former Union Minister of Finance in India. In October, Chidambaram was charged with corruption, forgery, and cheating by Indias Central Bureau of Investigations in October, and was granted bail in December. Chidambaram has denied wrongdoing.

Neda Ashtari, a second-year medical student at UCLA, stood on the stairs of Powell Library in October, dressed in all black.

In front of her were tens of white paper bags, each with a small plastic candle inside and a name scribbled on the front in colored marker. Students sprinkled plastic rose petals in between the bags, a sort of vigil for the lives lost as a result of UCLAs inaction to drop the patent on Xtandi, they wrote in a pamphlet.

Ashtari protested the patent appeal in India for years. In Regents meetings she stood in front of University of California leadership and demanded change.

Liz Ketcham

Can you imagine the guilt you would feel if all of your familys income went to your [medical] bills? Would you even want to live at that point? she asked. For one moment can you just step outside yourselves and imagine having to choose between the roof over your head and the (medicine) you needed to stay alive?

But on that day in October, standing in front of the bags of names, she seemed quieter more defeated than angry.

The pleading, public protests, and letters to the UC presidents officeone signed by 56 civil society organizations and doctors, then another with 3,500 signatures signed by students and advocacy groups had been mostly ignored.

To her, UCLAs choice to defend the Xtandi patent is personal. Her mother was diagnosed with breast cancer when she was four years old. For 12 years, as her mothers primary caregiver, she fought with insurance companies to get medication. She lost her house, her father left under the mounting stress over the cost of treatmentsand eventually her mother.

She says the worst part wasnt saying goodbye to her mom, it was knowing the same financial nightmare would happen over and over again.

They say time heals all wounds but it hasnt, she says, her voice breaking up. I still live with the consequences of these policies every day.

In a small coffee shop on the outskirts of New Delhi, Rupam Borah flips through pictures of his dogs on his phone.

Theres Bella, who he found badly beaten on the side of the road; Phi Phi, who he rescued on the way back from his vacation on Phi Phi Island, and Coco, whose paw was run over by a car.

He has a soft spot for stray dogs. He fosters stray puppies, pays to neuter dogs, and even puts out bowls of Purina dog food on the street in front of his house.

Feeding the dogs is getting harder. Paying for everything is getting harder.

Liz Ketcham

Rupams business has been slowly declining for months. When the economy is bad, the last thing people want to do is redecorate their houses, he says. Worried about the future of his company, he started teaching his employees the skills they would need to start their own businesses.

Regardless, he tries to stay positive, especially around his fatherhes seen too many children turn cold and resentful towards their parents under the financial strain of treatment.

He coos and zooms in on the well-groomed hounds as he goes through photos, occasionally stopping to take a sip from a clay cup of chai. In one, a dog with short white fur and pink ears is flashing a soft, squinty smile at the camera. In another, Rupam nuzzles into the dogs fur as the brown-and-white mutt playfully nibbles at the hand wrapped around him.

When he tells his story, he speaks calmly and matter-of-factly. If his mother were at the table, though, he wouldnt be able to keep it together, he said.

Hes wracked with guilt over the whole situationthat his oldest daughter walks dogs to help pay for her sisters tuition, that he had to take back money from his mother to cover the cost of medication.

Rupam doesnt blame his father for wanting the medicine, but he wouldnt choose enzalutamide for himself: I would opt for euthanasia any day.

RELATED: Developers Are Attempting to Evict an Elderly Cancer Patient from Her WeHo Apartment

Stay on top of the latest in L.A. food and culture.Sign up for our newsletters today.

Visit link:
UCLA's Fight to Patent a Life-Saving Cancer Drug Could Make the Medicine Virtually Unobtainable in India - LA Magazine

The Deadly Effects of Stress on Our Bodies – Patch.com

As we enter the New Year, health, wellness, and resolutions to eat better and get more exercise are priorities for most of us who have not yet made them permanent fixtures in our way of life. Whether you launch into 2020 with a keto or Mediterranean diet and/or join Orangetheory, Equinox or that cult (I mean gym) CrossFitor just subscribe to the burn more calories than you take in tried and true methodwe all start off with the best of intentions. In the last couple of years, the newest health threat has been sitting too muchin our offices and homesand we have seen the emergence of stand-up desks and "how many steps have you taken today" has become a normal part of everyday conversation. But maybe the most well-accepted cause and least understood "silent killer" that we collectively do a terrible job of managing is stress.

The human body functions best when it is in a state of homeostasisin a condition of balance, harmony, and stability. Our bodies have evolved to have a number of compensatory mechanisms to maintain a balance for pH, O2/CO2, electrolytes, glucose/insulin, immune response activation/de-activation, hormone levels, and many other physiological systems. When the homeostatic state for these physiological balances is thrown off due to genetic or environmental changes over time, they may eventually experience a number of problems in their mental and physical functions. This may result in a variety of symptoms from headaches, nausea, and exhaustion to more pronounced situations, such as high blood pressure, diabetes, auto-immune diseases, and cancer.

The medical community continues to elucidate the cause and effect relationship between bacteria, viruses, toxins and our genetics and how those lead to illness. Researchers and companies continue to develop new and better drugs, vaccines, medical devices, and even gene therapy and gene-editing technology to "fix us" when we get sick. But what about our "mind-body" connection? And no, that is not just a term used by alternative medicine charlatans. The connections between our physical and mental well being are well established if still poorly understood mechanistically. Psychosomatic Disorders are a set of physical diseases that are either caused or made worse by mental factors such as anxiety or stress. According to the Cleveland Clinic, depression can be caused by chronic illness and further exacerbate the illness thereby "causing a vicious cycle to develop."

The rates for depression that occurs with other medical illnesses is quite high (national average for depression without co-morbidities is ~7-10%):

A recent article in Psychology Today explains that stress is a constant in our lives; it is always in flux and that stress and our bodies' response to it is not in and of itself a bad thing. Think "fight or flight" our evolutionary response to making sure we didn't get eaten by that annoying dinosaur. That dino-stressor kicks off a cascade of hormones that elevate our blood pressure, heart rate and increase our energy burn presumably so we can outrun that T-Rex. That's all well and good, but I don't think I will need to outrun a dinosaur or even a lion anytime soon. So what if our bodies are exposed to other non-carnivore caused periodic high-level stressors or chronic state of stress that trigger these cascades? What does that do to our bodies?

In a recent review of the physiological effects of stress, scientists and clinicians have demonstrated the negative effects on everything, including our central nervous system, cardiovascular, immune, gastrointestinal and endocrine systems. We are all familiar with stress-induced headaches, difficulty sleeping, and upset tummies, but unmanaged stress can also negatively impact a person's cardiovascular system by increasing blood pressure and heart rate which over time can lead to "stress" cardiomyopathy or increased risk of heart attack and stroke. Studies have shown impairments in memory recall, as well as cognitive processing, are also linked to chronic stress.

Let's face it, we all encounter some form of stress usually on a daily basis. We have demanding workdays, tiresome and/or tedious long commutes to our jobs, worrying about bills and the demands and responsibilities that come with raising our kids. Speaking from experience, couples will also experience stress from their relationships at times and business owners can also be stressed out about the success of their personal enterprises. While we can all expect to be stressed out over something in our lives at some point, allowing ourselves to continually remain in a state of stress could lead to serious health problems.

Healthline reports people who are stressed usually have a hard time controlling their emotions. Sound familiar to anyone? These emotional outbursts can lead to or exacerbate already existing problems with our spouse (or significant other), friends, family or co-workers. This can lead to even more stress and physical illness.

So, as you make those New Year's resolutions, let's not lose sight of how important removing or managing our stress is to our health, happiness, and well being. Resolve to try to be more self-aware of your own stress and how it is affecting you and others around you. Don't be afraid to talk to your doctor, spouse, family or friends about things that are stressing you. There is no shame in speaking up and you can take comfort in the knowledge that you are definitely not alone. Take steps to remove stressors where you can. When that is not possible, identify and try a variety of ways to cope with, manage and reduce that stress. Look, I am not an expert on what works or doesn'tdifferent things work for different people. Maybe it's a long walk, listening to music, meditating, hitting the gym, playing a video game with your kids or just giving and receiving a BIG hug from your spouse.

Life is life and we will never be stress-freebut let's resolve to reduce our stress levels in 2020 and to help those around us as well.

Originally published at erikhalvorsen.org on January 9, 2020.

More:
The Deadly Effects of Stress on Our Bodies - Patch.com

Conducting Gene Therapy in Colorado – dujour.com

Im not sure how the task fell to me, but over the years I have become my familys de facto genealogist. To my amateur sleuthing credit, I have uncovered more secrets and put to bed more family rumors than one could imagine. During my quest to learn more about my family heritage, I purchased a 23andMe genetic test, which can tell you not only the geographical roots of your human existence but also your migration routeand even genetic traits like the fear of public speaking. What I didnt realize at the time was the treasure trove of information that existed on the back end of a 23andMe test, information that may be even more important to my well-being and that of my descendants than knowing the likelihood that I move more than average during sleep.

This rich data is already collected as a part of these tests but is not directly reported to the user. It contains information on vulnerabilities in nutrition, inflammation, and detoxification capacity; susceptibility to heart or neurological disease, cognitive or mood dysfunction, and cancer; and deficiencies in cellular metabolism, including which nutrients are in higher demand. This is the kind of imperative knowledge I wanted to have, so I set out to find a professional to analyze it.

The journey took me to the Strata Integrated Wellness Spa at Garden of the Gods Resort & Club in Colorado Springs, Colorado, where I checked into one of its new casitas for a long weekend. There, Karly Powell, a registered naturopathic doctor with expertise in therapeutic nutrition and functional biochemistry, has developed a program called Decode Your DNA. In it, she conducts a specialized analysis of the raw data of a clients DNA to provide an understanding of an individuals genetic health background and deficiencies and then creates a personalized wellness plan.

According to Dr. Michael Barber, Stratas medical director, the most common issues are found are in the so-called methylation pathways, which control how our bodies handle nutrients, supplements, and medications. Abnormalities in these pathways can influence the risks for neurological, cardiac, cardiovascular, gastrointestinal, and other systemic illnesses. With my newly analyzed DNA information, Powell crafted a custom-made, gene-based prescription for me that included a long-term dietary program, targeted nutritional supplementation, and lifestyle modifications.

That weekend, I began to make her suggested modifications and utilized the doctors, physicians, clinicians, and therapists at Strata. Strata is one of the few spas in the country that offer this kind of personalized medicine paired with bespoke nutrition and fitness, as well as the opportunity to address cardiology, kinesiology, and energy needs in the same facility.

Many people say that your genesgeneticsare not your future but your potential, Barber says. Knowing in advance where one might be susceptible to issues such as heart disease, cognitive or mood dysfunction, cancer, or nutritional issues may allow a person to alter critical components of diet, exercise, [and] lifestyle to maximize their potential for optimal health.

See the article here:
Conducting Gene Therapy in Colorado - dujour.com

The Region’s Health Leaders on What’s New and What the Next Decade Will Bring – Business West

Vision 2020

Few industries change as rapidly and as dramatically as the broad, multifaceted realm of healthcare. From oncologists use of cancer fingerprinting and gene therapy to facial transplants for accident victims; from cutting-edge protocols to save the lives of stroke and heart-surgery patients to a dizzying array of new treatments to improve vision the list is seemingly endless, making it impossible to paint a full picture of where healthcare has come in the past decade.

But we at BusinessWest wanted to try anyway and, at the same time, look ahead at what the next decade might bring. So, appropriately, here at the dawn of 2020, we invited a wide range of healthcare professionals to tell us what has been the most notable evolution in their field of practice in the past 10 years, and what they expect or hope will be the most significant development to come in the next decade.

The answers were candid, thoughtful, sometimes surprising, but mostly hopeful. Despite the many challenges healthcare faces in these times of advancing technology, growing cost concerns, and demographic shifts, the main thread is still innovation smart people working on solutions that help more people access better care. After all, healthcare is, at its core, about improving peoples lives, even when they seek it out during their direst moments.

Innovation and promise. Thats what we believe a new decade will bring to all corners of the healthcare world that is, if these leaders, and countless others like them, have anything to say about it.

Joanne Marqusee

President and CEO, Cooley Dickinson Health Care

Joanne Marqusee

The most significant recent development in healthcare administration has been a recognition of the role patients play in their own healthcare. Crossing the Quality Chasm: A New Health System for the 21st Century, published in 2001 by the Institute for Healthcare Improvement, called for a massive redesign of the American healthcare system. Specifically, it provided Six Aims for Improvement, five of which focused on safety, effectiveness, timeliness, efficiency, and equity. Not talked about as much, the sixth aim was to make healthcare patient-centered.

While we still have a ways to go to truly be patient-centered, we have witnessed a sea change in the past decade in this regard. Patients are increasingly active participants in their care, questioning their doctors and other providers to ensure that they understand their options, using electronic medical records to engage in their care, and speaking out about what they want from treatment or forgoing treatment at the end of life. The best healthcare providers both organizations and individuals embrace these changes, welcoming patients as more than recipients of care, but rather active partners in their own care and decision making.

My hope for the most significant development over the next decade has to do with providing universal healthcare coverage while controlling healthcare costs. While we almost have universal coverage in Massachusetts, too much of the nation does not. A hotly debated topic, universal healthcare has many benefits, including increasing access to preventive and routine medical care, improving health outcomes, and decreasing health inequalities.

Dr. Nicholas Jabbour

Chairman, Department of Surgery, Baystate Medical Center

Dr. Nicholas Jabbour

The most significant development in surgery over the past decade has been the move toward less invasive surgical approaches made possible through advanced technology. These approaches include robotic and minimally invasive surgery, including intraluminal surgery in areas such as gastroenterology, cardiology, and neurosurgery for exemple, the passage of an inflatable catheter along the channel inside of a blood vessel to enable the insertion of a heart valve instead of making a large opening in the chest. As a result, we have seen a big shift from inpatient to outpatient surgery with shorter hospital stays and improved post-op recovery.

In the next decade, we foresee these innovations in less invasive surgery will be enhanced by better computing and software integration. This interaction will include the merging of radiological and potentially pathological information which is currently available in a digital format with real-time visualization of anatomical structure during surgery. This will offer surgeons the opportunity to improve the accuracy and speed of a surgical procedure while minimizing the risks.

The next decade will also see major innovation in the area of transplantation with the development of tissues or whole organs through bio-engineering manipulation of animal or a patients own cells. The integration of this bio-engineering manipulation with currently available technology, such as 3D printing and 3D imaging, will provide patients with the needed tissue or organ including valves, bone grafts, hernia mesh, skin, livers, and kidneys in a timely manner. This development will revolutionize the field of transplantation and surgery in general.

Karin Jeffers

President & CEO, Clinical & Support Options Inc.

Karin Jeffers

Over the past 10 years, weve seen a growing adoption within the behavioral-health and medical fields of holistic treatment models. While the two disciplines were once treated as different animals, the entire health field is now moving to treat both the body and the mind together. The next 10 years are likely to bring these two fields even closer.

Today, youre seeing behavioral-health clinicians being hired into physical health practices. Likewise, physical health providers are cross-training to better understand behavioral issues. Whereas, a decade ago, a behavioral-health client might be assigned a therapist or a psychiatrist, they are now gaining access to more robust set of supports, including nursing, case management, recovery coaching, and peer support from those with lived experience. Government mandates and payment model changes are forcing outcomes-based integration, too. Pediatricians, for example, must now do behavioral-health screenings of all youth under 21. In the mental-health space, youre seeing clinicians ask about weight, exercise, and other physical factors.

Were seeing significant movement on both the state and federal levels to value outcomes over volume. Its reflected in the criteria set by the Excellence in Mental Health Act for certified community behavioral-health clinics, a designation CSO has earned, and in the work we have done with the Substance Abuse and Mental Health Services Administration. Our ability to tailor programs, like our grant-funded work at the Friends of the Homeless shelter in Springfield, has literally saved lives among those experiencing homelessness and co-occurring conditions, like substance-use disorders.

In the coming years, we hope to see integrated care models become even more mainstream. Things appear headed in the right direction, but government action establishing payment reform within the behavioral-health field needs to be taken and the integrated models need to be appropriately funded. Such changes would affirm overall health and wellness to include both physical and behavioral health.

Dr. Yannis Raftopoulos

Director, Holyoke Medical Center Weight Management Program

Dr. Yannis Raftopoulos

Weight management is a rapidly evolving field, and I am fortunate to be part of it. One of the most significant innovations this field has experienced in the last 10 years was the development of a new gastric balloon. Packaged in a small capsule and swallowed with water, the Elipse balloon provides satiety while requiring no procedure or anesthesia for its placement and removal. Together with its excellent safety profile, the Elipse balloon is the least invasive and yet effective weight-loss modality available today. Elipse is manufactured in Massachusetts by Allurion Technologies.

I had the opportunity to be an investigator in the European trial which led to the Elipse market approval in the European Union in 2016. Recently, Holyoke Medical Center was among 10 U.S. sites in which an FDA-regulated trial was conducted. The trial was completed successfully, and Allurion has submitted data requesting FDA approval to market Elipse in the U.S. The balloons use in Europe shows that patients can lose more than one-fifth of their initial weight.

A New England Journal of Medicine study reported that 107.7 million children and 603.7 million adults, among 195 countries, were obese in 2015. High body-mass index accounted for 4 million deaths and contributed to 120 million disability-adjusted life-years. Obesity is a chronic disease, and its management requires long-term guidance and close patient-physician communication. Successful collaborations between existing best practices with technology innovations that will allow delivery of effective weight-management care on a massive and global scale could be the most significant evolution in the field in the next 10 years.

Dr. Hong-Yiou Lin

Radiation Oncologist, Mercy Medical Center

Dr. Hong-Yiou Lin

The advent of new medical oncology drugs has improved control of microscopic and, to a lesser extent, macroscopic disease, allowing local treatments, such as surgery or radiotherapy, to increase survival. To cure cancer, we need to eliminate cancer cells where they started, as well as any microscopic cells traveling through the body. The idea of using immunotherapy to fight cancer has been around for decades, but bringing this idea to the clinic has been hampered by the cleverness of cancer cells knowing how to evade detection by our immune system. Recently FDA-approved immunotherapy either takes away that invisibility cloak or wakes up our dormant immune cells to start fighting cancer.

The biggest development in oncology in the next 10 years will be personalized precision medicine, which allows the oncology team to tailor treatment to each patients unique cancer biology and life circumstances. Meanwhile, improvements in cancer diagnosis will come from novel PET radiotracers and new MRI sequences that allow for more accurate staging and identification of the best site to biopsy. Pathologists will use novel tools such as genome sequencing to supplement traditional microscopy to subclassify the specific type of cancer within a certain diagnosis instead of grouping into broad categories.

Surgical, medical, and radiation oncologists can then use the above information to decide on the best sequencing between surgery, systemic therapy, and radiotherapy to minimize side effects and maximize cure. Medical oncologists will be able to offer more drugs that target new mutations, overcome drug resistance, increase specificity to a mutation, or better fine-tune immunotherapy, targeting only cancer cells by enlisting gene modification as well as natural killer cells. Radiation oncologists will have new radiomic and genomic tools to personalize the radiation dose and volume, and when to offer radiotherapy.

In short, over the next 10 years, cancer care will continue to move away from the traditional one-size-fits-all model toward a more personalized approach.

Dr. Jonathan Bayuk

Medical Director, Allergy & Immunology Associates of New England

Dr. Jonathan Bayuk

There have been incredible and exciting advances in allergy and immunology in the last two years. However, the unmet needs of allergic and autoimmune-disease-afflicted patients has grown dramatically in the last 20 years. In response to the increasing prevalence and acuity of allergic diseases and autoimmune diseases, the world has launched products to help address these very severe patients. These medications are indicated for many conditions and work very well. They are generally safe, but are very expensive. These medicines are different than traditional pharmaceutical drugs as they are not chemicals, but biologically derived medicines designed to augment or modify the immune response. As such, they are call biologic medications.

In the field of allergy and immunology, we can now dramatically treat and potentially cure many diseases that in the past were very challenging to manage. The biologic medicines that we have now treat asthma, eczema, allergic disease, and hives. The patient selection is based on severity of their condition, and these medicines are only for moderately to severely affected people. If, as a medical profession, we were to place as many people as possible on these therapies, the cost would be astronomical and not sustainable.

However, is it fair to deny any of these patients access to these treatments who truly need them? I would argue that choice is a very difficult one to make, and as physicians, our primary goal is healing at whatever cost. As a nation, we have a dilemma. Can we afford the medicines we have or not? It is unclear that any serious legislative body is willing to tackle that question. For now, the use of these medicines is changing lives dramatically, and it is an exciting time to be able to use these newer tools to help our patients live better lives.

Dr. David Momnie

Owner, Chicopee Eye Care

Dr. David Momnie

What are the most significant advancements in eye care in the last decade? It depends on whom you ask. Retinal ophthalmologists would probably say its the treatment of wet macular degeneration, a leading cause of blindness, with anti-VEGF injections. Cataract surgeons would most likely cite small-incision surgery and new lens implants that often leave patients with 20/20 vision. Glaucoma specialists might tell you its the development of MIGS, or minimally invasive glaucoma surgery. These operations to lower the pressure in the eye use miniature devices and significantly reduce the complication rate.

Primary-care optometrists and ophthalmologists would no doubt talk about the advances in optical coherence tomography, a remarkable instrument using light waves that gives cross-sectional pictures of the retina. The technique is painless and non-invasive and is becoming the gold standard in eye care because it has revolutionized the diagnosis and treatment of glaucoma and macular degeneration. For optometrists specializing in contact lenses, using newly designed scleral lenses to restore vision in people with a corneal disease called keratoconus has been a major development. There are many other specialists in eye care, including LASIK surgeons, that have seen remarkable changes in technology.

What will the next decade bring? Artificial intelligence (AI) is becoming more accurate for screening, diagnosing, and treating eye conditions. AI systems can increasingly distinguish normal from abnormal pictures of the retina. Where there is a shortage of ophthalmologists and optometrists, AI screenings combined with telemedicine, providing remote care using communications technology, may be able to find and treat more people who are falling between the cracks of our healthcare system. The term 20/20 is the most common designation in eye care, and the year 2020 will probably usher in another decade of remarkable developments in our field.

Teresa Grogan

Chief Information Officer, VertitechIT

Teresa Grogan

From the perspective of technology that enables healthcare, the biggest game changer of the last decade has been the iPhone and now, essentially any smartphone.

Steve Jobs introduced the first iPhone in 2007 (a little over a decade ago), and physicians embraced it quickly. It started as a simple tool for doctors (applications like the PDR, or Physicians Desk Reference) for looking up drug interactions. Today, its a portable EMR, a virtual visit facilitator, and a remote-monitoring device for many healthcare providers, as many patients have embraced and insisted on this technology to improve access to care. As the cost decreases and cellular bandwidth improves, the rapid growth of the IoMT (Internet of Medical Things) will place smartphones at the center of the next wave of healthcare technology breakthroughs.

Looking forward, Id like to see complete elimination of passwords to access electronic information. While there has been some movement toward this with tap and go badges and fingerprint readers, a single standard is needed that would work regardless of the software program used. I hope there are greater strides in the creation, deployment, and adoption of other biometric technologies, like iris, face, or voice recognition, so that a healthcare professional could walk into a patient room or into a hospital and the computer systems would know his or her identity in immediate and secure fashion. If access to the data needed by a healthcare provider were as easy as turning on a light switch, the improvements in quality of life and efficiency in work for that provider would translate to improved patient outcomes.

Dr. Aaron Kugelmass

Vice President and Medical Director, Heart and Vascular Program, Baystate Health

Dr. Aaron Kugelmass

We have seen many improvements in cardiovascular care over the last 10 years, but the development, approval for clinical use, and dissemination of transcutaneous aortic valve replacement (TAVR) stands out as the most dramatic. This new technique allows cardiologists and cardiac surgeons, working together, to replace the aortic valve without opening a patients chest or utilizing heart-lung bypass, which has been the standard for decades. This less invasive approach is typically performed under X-ray guidance and involves accessing a blood vessel in the leg and guiding a catheter to the heart.

The TAVR procedure was first approved for clinical use in November 2011. It was initially limited to very sick patients, who were not candidates for traditional surgery because of the risk it posed to them. TAVR allowed patients who otherwise could not receive life-saving valve surgery to have their valves replaced with improvement in longevity. With time and experience, the procedure was approved for lower-risk patients as well, and more recently has been approved for the majority of patients, including those with low operative risk. TAVR has been shown to be equivalent or safer than traditional aortic valve-replacement surgery, and is quickly becoming the procedure of choice for most patients who require an aortic valve replacement. Since the procedure typically does not require open-heart surgery, recovery time is much shorter, with some patients going home within a day or two.

In the next 10 years, we expect that similar less-invasive procedures with shorter recovery time will be developed for other heart-valve conditions in patients who otherwise could not receive therapy.

Beth Cardillo

Certified Dementia Practitioner and Executive Director, Armbrook Village

Beth Cardillo

During the last 10 years, neuroscientists have been researching the causes of Alzheimers disease. There has been much discussion about which comes first the amyloid plaque or the fibrillary tangles that develop in the brain, which are roadblocks to cognition, thus causing the difficulties with Alzheimers and other related dementia. That question has not been answered yet. Researchers were able to isolate the APOE gene, which is a mutant gene that is found in familial Alzheimers disease, helping us to better diagnose it. We have also better understood how diet, exercising both body and brain, and lifestyle contribute to the disease. Currently there are 101 types of dementia, with Alzheimers accounting for 75% of cases.

The next 10 years will result in more preventive actions. One major action will be to help people avoid developing type 2 diabetes, which may be labeled the next cause of Alzheimers (this type of Alzheimers is already being called type 3 diabetes). There has been a major link between sugar in the hippocampus and Alzheimers disease. Though there is no cure yet for Alzheimers, we are finding more information based on genetics, diet, and PET scans, which can show shrinkage in the brain.

Every year, researchers are more hopeful that a new drug will be developed to eradicate the disease. The last new drug from Biogen was looking hopeful in clinical trials, but that turned out to be not the case. Prevention continues to be at the forefront, as well as participating in clinical trials. More people who do not have dementia or mild cognitive impairment are desperately needed for clinical trials so comparisons of the brain can be made.

Ellen Furman

Director of Nursing, American International College

Ellen Furman

As in all healthcare, the one thing that can be ascertained is constant change. The same can be said in nursing education today. No longer is the instructor-led lecture method of teaching considered best practice in education, but rather the shift to using class time to apply learned concepts. One way this is done is through the flipped classroom. Using this educational modality, students study the concepts being taught preceding the class, followed by class time where students apply these concepts in an interactive activity, thereby developing students abilities to think critically, reason, and make healthcare judgements based upon the application of knowledge.

Another change in nursing education is an expanded focus away from pure inpatient (hospital-based) clinical education to outpatient (community-based) clinical education. While hospital-based education remains essential, the realization that most healthcare provided is in outpatient settings has broadened the clinical experiences required to prepare the graduate registered nurse for care provision.

Additionally, with healthcare as complex as it is, nursing students are being taught to be prepared for entry into practice. Education regarding the use of evidence-based practice, how to apply for the licensure examination, preparation to be successful on the National Certification Licensure Exam, nurse residency opportunities, interviewing techniques, transitioning from student nurse to registered nurse, etc. are all taught using a variety of educational modalities based upon the current best available evidence in nursing education.

As we forge ahead in healthcare, nurse educators will continue to evolve to meet healthcare needs through the education of nursing students so as to prepare them to provide care to meet the needs of those we serve well into the future.

James Haas

Co-owner, Orthotics & Prosthetics Labs Inc.

James Haas

Advances in prosthetic technology have clearly been the most significant development in my field over the past decade. From knees and feet that adapt to different walking speeds and terrains to hands that send sensations of touch to the brain, every aspect of patient care has changed and continues to change at a rapid pace.

Prosthetic feet, knees, and sockets have been greatly impacted. Once made from multi-durometer foams and wood, the prosthetic feet of today are made from carbon, fiberglass, and kevlar laminated with modified epoxy resins. They store energy and adjust to uneven terrain and hills. Microprocessor knees have on-board sensors that detect movement and timing and then adjust a fluid/air control cylinder accordingly. These knees not only make it safer for a person to walk, they also lower the amount of effort amputees must use, resulting in a more natural gait. Sockets once made from stiff materials are now incorporated with soothing gels and flexible adjustable systems that allow a patient to make their own adjustments to improve their comfort.

As for the next decade, I hope to see national insurance fairness. Devices typically last about three to five years. Some people make them last longer, but others, especially growing children, need replacements more often. Many private insurance plans have annual caps and lifetime limits on coverage for orthotics and prosthetics. The Amputee Coalition of America authored insurance-fairness legislation and has lobbied for its implementation for over a decade. This legislation has been ratified in 20 states, including Massachusetts. The Fairness Act requires all insurance policies within the state to provide coverage for prosthetics and orthotics equal to or better than the federal Medicare program and have no coverage caps and lifetime restrictions.

Dr. Lisa Emirzian

Co-owner, EMA Dental

Dr. Lisa Emirzian

The most significant development in the field of dentistry over the past decade has been the integration of digital technology into our daily practices. There are three components of digital dentistry: data acquisition, digital planning, and, finally, the manufacturing of the restoration to be created. Data acquisition today is accomplished with digital radiographs, paperless charting, intra-oral scanners, cone-beam 3D scanners, and video imaging. For the planning process, we now have the ability to merge the data with software that enables computer-aided design and digital smile design, allowing dentists to perform complex procedures, including guided surgical treatments and smile designs, with optimum results. Fabrication and execution of the final restorations can be done in the office or, more often, in laboratories with highly sophisticated digital milling machines, stereolithography, and 3D printing.

In the next decade, we will see data fusion to ultimately create the virtual patient. The next-generation digital workflow will merge intra-oral 3D data with 3D dynamic facial scans, allowing dentists to create 3D smile designs and engineer the dentofacial rehabilitation. The integration of scanners and software will expedite the delivery of teeth in a day. In addition, multi-functional intra-oral scanners will allow for early detection of carious lesions and determine risk levels for different patients.

Above and beyond this foreseeable future, artificial intelligence (AI) will be the next paradigm shift. Companies are already looking for big-data collection and deep machine learning to help the practitioner in their everyday chores of diagnosis and treatment. AI cloud-based design platforms will input data, and AI engines in the background will aid in all parts of dental treatment, including diagnosis, design, and fabrication of final restoration.

Let us not forget one thing: the future is all about us people utilizing technology to enhance the human connection between doctor and patient.

John Hunt

CEO, Encompass Health Rehabilitation Hospital of Western Massachusetts

John Hunt

A significant rehabilitation development from the past includes one that may surprise you. Time. A luxury we once knew, time meant patients could recover in a hospital longer after a surgery, an accident, or an illness. Nurses had more time to assess patients to know exactly what they needed. Insurance companies approved longer patient stays through lengthy consideration. Ten years ago, a stroke survivor could recover for two weeks in a hospital and then join us for a rehabilitation stay that would last several weeks.

Today, a three- to five-day stay in the referring hospital, followed by a two-week stay in rehabilitation, is the norm. We are seeing significant decreases in the age of stroke survivors as well as an increase in the number patients who survive with cognitive and physical disabilities. Yet, we also see medical breakthroughs, including the discovery of tissue plasminogen activator (TPA) nothing short of a miracle. TPA actually reverses the effects of an evolving stroke in patients when used early on, making recoveries easier.

With new advanced technologies being introduced every year, rehabilitation continues to progress at a rapid speed. Looking into the future, evidence-based research will continue to grow to help us make knowledgeable decisions that ultimately impact patient outcomes. Increased clinical expertise will lead to higher functional gains in shorter amounts of time. As a result, acute inpatient rehabilitation will impact the lives of patients like weve never seen before.

Dr. Susan Bankoski Chunyk

Doctor of Audiology, Hampden Hearing Center

Dr. Susan Bankoski Chunyk

The most common treatment for hearing loss is hearing aids. Although digital processing has been available in hearing aids since 1996, the past 10 years have offered great leaps in technology for people with hearing loss. Each generation of computer chip provides faster and smarter processing of sound. Artificial intelligence allows the hearing-aid chip to adjust automatically as the listening environment changes, control acoustic feedback, and provide the best speech signal possible. People enjoy the convenience of current hearing aids Bluetooth streaming, smartphone apps, and rechargeable batteries.

These features are the icing on the cake, but the real cake is preservation of the speech signal, even in challenging listening situations. Since the primary complaint of people with hearing loss is understanding in noise, new hearing-aid technology works toward improving speech understanding while reducing listening effort in all environments. This significantly improves the individuals quality of life.

The negative effects of untreated hearing loss on quality of life are well-documented. Recent research has also confirmed a connection between many chronic health conditions including diabetes, cardiovascular disease, kidney disease, balance disorders, depression, and early-onset dementia and hearing loss. This research shows that hearing loss is not just an inevitable consequence of aging, but a health concern that should be treated as early as possible. My hope for the future is that all healthcare providers will recognize the value of optimal hearing in their patients overall health and well-being and, just as they monitor and treat other chronic health conditions, they will recommend early diagnosis and treatment of hearing loss.

Continued here:
The Region's Health Leaders on What's New and What the Next Decade Will Bring - Business West

Meet the St. Baldrick’s Foundation 2020 Ambassadors – PRNewswire

LOS ANGELES, Jan. 7, 2020 /PRNewswire/ --Seeing the world through the eyes of a child is often filled with wonder and imagination. It should not include things like hospital beds, needles and chemotherapy. Sadly, these things are a reality for kids with cancer. To raise awareness about childhood cancers and the need to fund research to find cures and better treatments, the St. Baldrick's Foundation, a non-profit on a mission to defy childhood cancers, introduces its 2020 Ambassadors. The five Ambassador families, each touched by childhood cancer, will share their journeys of struggle and triumph, hope and despair, and give people a glimpse into their lives and what comes after hearing those life-changing words, "Your child has cancer."

Coming from all areas of the country, ages, disease types and stages of their cancer journey, the Ambassadors represent the more than 300,000 kids diagnosed with cancer each year worldwide and serve as a reminder that cancer doesn't discriminate.

This year's Ambassadors are:

Seth and Joel Deckerare identical twins with a very special bond not even cancer could separate. The twins did everything together. Along with their older brother, Nathaniel, they filled the Decker home with the sound of laughter and play. Seeing the boys push vehicles around and make loud noises or roar and stomp around pretending to be dinosaurs were familiar sights. But in an ironic twist of fate, the twins were both diagnosed with cancer: Seth was diagnosed with a rare form of acute myeloid leukemia (AML) in December 2016 and three months later, a biopsy revealed Joel also had AML. With the overlapping diagnoses and treatments, the family endured months of separation and treasured the few weeks they could be together at home. Despite both boys receiving bone marrow transplants and enduring complications from the procedures, they relapsed. Surrounded by their loving family, Joel died November 2017 at the age of 3, followed by Seth in May 2019 when he was 4 years old. The Decker family firmly believes much more research is needed for AML, especially when the disease has relapsed. They have created a St. Baldrick's Hero Fund in memory of Joel and Seth to support research that will help find new treatments and cures so other families won't have to say goodbye too soon.

Hudson Walker, 1, from Englewood, Colo., was diagnosed with Ewing sarcoma in February 2019, a rare disease that makes up only 2 percent of all childhood cancers and is very uncommon in babies. Chemotherapy began right away and surgery to remove her scapula resulted in the good news of clean margins. Hudson had a positive attitude through it all, even looking forward to going to the hospital to see the doctors, nurses and therapists who had become her friends. While happy she is cancer free after treatment, her parents know that her cancer journey is not over. As she grows, they will need to remain vigilant with monitoring potential late effects from chemotherapy. But for now, Hudson is enjoying each day, loving her family and her favorite things: queso, french fries, the color pink, unicorns, the movie Aladdin, and all kinds of music especially Lizzo and Kacey Musgraves. Armed with her sparkling personality and the bravery many of us only dream of, Hudson's family is confident she can take on any challenge life puts in her path.

Micah Bernstein, 9, from Carlsbad, Calif., has spent the bulk of his life fighting cancer. Micah was diagnosed with neuroblastoma in March 2012 at just 15 months old. He's had three surgeries, 21 cycles of chemotherapy and 36 sessions of radiation. Two separate phases of his treatment were centered around Unituxin, an immunotherapy drug developed with support from St. Baldrick's that received FDA approval while Micah was in treatment. Since then, Micah's blood work and scans have been clear for more than five years and he's been completely off treatment for nearly a year, which means an important milestone for Micah his first survivorship clinic visit in 2020. Micah is very interested in science and wants to become a doctor. He even has a message for researchers involved with St. Baldrick's: "Thank you for creating new medicines for kids with cancer. Those medicines save kids' lives, and one of them saved mine." Micah's parents, Jeff and Kate, are very passionate about helping to fund the most promising childhood cancer research and have established a St. Baldrick's Hero Fund, the Mighty Micah's Mission Fund, aiming to raise at least $100,000 to fund neuroblastoma research.

Austin Schuetz, 11, from Fall River, Wis., was diagnosed with a high-risk form of acute lymphoblastic leukemia just before his third birthday. Austin faced 3 years of treatment including intense chemotherapy, bone marrow biopsies, and six days of daily cranial radiation. Before Austin could finish treatment, he relapsed. At that point, a bone marrow transplant was his only option for a cure. When that didn't work to eradicate the leukemia, Austin needed a miracle. That miracle came in the form of a gene therapy that uses a child's own immune system to fight the cancer. Austin was accepted in a clinical trial, supported by the St. Baldrick's Foundation, that would collect his T-cells in a lab and train them to seek out and kill the cancer cells. The treatment worked, and Austin is now six years out from the clinical trial that saved his life. Because of research, Austin can enjoy the things he loves, like video games, basketball and Nerf guns.

Shamari Brazile, 14, from Cleveland Heights, Ohio, was diagnosed with osteosarcoma when she was 13 years old. In November 2017, during the middle of basketball season, she complained of pain in her right hip. When the pain didn't improve, tests revealed a mass on her pelvis. Shamari was diagnosed with osteosarcoma in March 2018 and started treatment right away. She endured 10 weeks of inpatient chemotherapy and surgery to remove the tumor, followed by 18 more weeks of chemotherapy before finishing treatment in December 2018. Less than three months after her last treatment, Shamari was already back on the lacrosse field, playing her favorite sport. In her free time, she also enjoys hanging out with friends, listening to music, drawing, teaching herself how to play the ukulele and writing short stories.

St. Baldrick's Ambassadors and their families will act as spokespeople for the St. Baldrick's Foundation, attend events and fundraisers and share their stories to educate the public about the realities of childhood cancers.

Continue following these six courageous storieson the St. Baldrick's blogand social media channels: Facebook,Twitter,YouTubeandVimeo. To learn how you canget involvedvisitwww.StBaldricks.org.

About St. Baldrick's FoundationAs the largest private funder of childhood cancer research grants, the St. Baldrick's Foundation is leading the charge to take childhood back from cancer. St. Baldrick's funds some of the most brilliant childhood cancer research experts who are working to find cures and better treatments for all childhood cancers. Kids need treatments as unique as they are and that starts with funding research just for them. Join us at StBaldricks.org to help support the best childhood cancer research, no matter where it takes place.

SOURCE St. Baldrick's Foundation

http://www.stbaldricks.org

See the original post:
Meet the St. Baldrick's Foundation 2020 Ambassadors - PRNewswire

Gene editing breakthroughs that cured genetic diseases in 2019 – The Star Online

IN the summer of 2019, a mother in Nashville, Tennessee in the United States, with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old said.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so that doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as a molecular scissors.

The genetically-edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary, but theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease beta thalassemia.

She had previously needed 16 blood transfusions per year. Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible.

It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the co-founder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Gene cures

Crispr was the latest breakthrough in a year of great strides in gene therapy, a medical adventure that started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not, such as making normal red blood cells in Grays case or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he said. It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the US National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said US NIHs associate director for science policy Carrie Wolinetz.

These therapies are exorbitantly expensive, however, costing up to US$2 million (RM8.18 million) meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to Massachusetts Institute of Technology (MIT) researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

In this Oct 10, 2018, photo, He speaks during an interview at his laboratory in Shenzhen, China. The scientist was recently sentenced to three years in prison for practicing medicine illegally and fined 3 million yuan (RM1.76 million). AP

Bioterrorism potential

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

In 2018 in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA (deoxyribonucleic acid) of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV (human immunodeficiency virus), even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, he added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species, e.g. malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist -- weve been talking about bioterrorism for years, she said. Nothing has ever happened. AFP Relaxnews

Read the rest here:
Gene editing breakthroughs that cured genetic diseases in 2019 - The Star Online

Perils of Project Nightingale – Ophthalmology Times

Abstract / Synopsis:

Google-Ascension deal ignites private data debate

It is a capital mistake to theorize before you have all the evidence. Sherlock Holmes, A Study in Scarlet

I was invited to participate in a recent panel about emerging trends in the future of healthcare. Artificial intelligence, telemedicine, gene therapy, and immuno-oncology are all fascinating scientific advances that people like to think about.

As we enter another election cycle, some politicians are calling for dramatic overhauls of the U.S. healthcare system. This includes price controls and European-style, mandatory single-payer governmental insurance (Medicare for All), which suggest the possibility of enormous change in how this huge industry works.

Previously by Dr. McDonnell: The problem with patient reviews

The panel discussion was moving along in a reasonable way and the audience of a few hundred seemed to be listening closely. Each panelist would address the question asked of him or her and the room was otherwise fairly quiet. If the moderator or a panelist inserted a little humor into the discussion, the audience would laugh politely, but that was about it.

Then a question was asked of one of my fellow panelists: There is tremendous interest in many sectors (insurance companies, pharmaceutical companies, researchers, etc.) in obtaining and analyzing the healthcare data of large numbers of patients. My question to you is: Who owns that data?

The panelist, an executive of a medical device manufacturer, responded quickly. Patients own their data. After a one-second pause, the audience, previously quiet, burst into loud applause.

People clearly care a great deal about this issue, was my immediate thought.

Related: Hospital closures hurt

The recent revelation about the existence of Project Nightingale and the ensuing uproar were both interesting and predictable. The Wall Street Journal reported that Google began the project in secret last year with St. Louis-based Ascension, a Catholic chain of 2,600 hospitals, doctors offices and other facilities and the second largest health system in the United States.

The data involved in the initiative encompasses lab results, doctor diagnoses and hospitalization records, among other categories, and amounts to a complete health history, including patient names and dates of birth. Neither patients nor doctors have been notified. At least 150 Google employees already have access to much of the data on tens of millions of patients.

As I understand it, based on news reports I have read, Ascensions position maintains that analyzing the data using artificial intelligence and machine learning will result in strategies to ultimately improve care of patients. The assertion is that the use of the data for this purpose is legal and ethical, and the companies did not need to secure permission from patients to start mining the data and did not need to inform its doctors that this huge data mining project was under way.

Not everyone else is so sure. According to Ellen Clayton, professor biomedical ethics at Vanderbilt University, the optics are bad.

The legal argument is tenuous, she said. Ethically, this is a bad strategy. They need to tell people what they are doing.

Related: How AI benefits patients and physicians

U.S. senatorsincluding Sen. Bill Cassidy, a Louisiana Republican who is a physicianare expressing concern about the program, calling for a moratorium or investigation or proposing legislation.

Again, according to The Wall Street Journal, Google wouldnt disclose the financial terms of the deal with Ascension. Nor would it say who at Google is allowed to access the data.

Lets presume the motives of all involved in Project Nightingale are pure. That does not change the fact that Americans do not want their data shared in secret deals. Having this program come to light this way was a mistake by both corporations.

Read more editorials here

References:

1.Googles Project Nightingale Gather Personal Health Data on Millions of Americans. WSJ. Nov 11, 2019

Original post:
Perils of Project Nightingale - Ophthalmology Times

Will 2020 be the Year of Telemedicine? – Medical Tech Outlook

Besides telemedicine, other technologies like wearables, robotic surgery and cutting-edge genomic technology will continue to trend in 2020.

Fremont, CA: Telemedicine will be the root cause for the next level of development in the industry. It addresses the basic need of consumers who crave for convenience. The driving force behind ride services like Uber and online shopping from Amazon also come home for health care technology stocks in 2020.

Imagine a scenario where a patient suffers from a severe illness. Telemedicine allows the patient to interact with the doctor virtually from home without needing an appointment. Telemedicine is taking off in a big way with which medicine and care have become a service. They provide the technology to enable remote care. An example would be a remote doctor and patient visit over the phone or internet via videoconferencing. This technology can also lift other existing healthcare technology services like medicine delivery, videoconferencing therapy options and many more. No doubt that health care technology companies will benefit from the trend.

There is still room for other technologies like wearables, robotic surgery and cutting-edge genomic technology. Wearables include the health tracking watches that continuously monitor patients health parameters. Meanwhile, related health care technology devices like WiFi-enabled scales and food-tracking apps are feeding directly into certain wearables. It is evident when patients with diabetes see better glucose-monitoring devices and insulin pumps in the same vein.

Intuitive Surgical leads the robotic surgery space, but also it faces rivalries from other health care technology companies. Several companies are already getting into this space, and still, there is a huge opportunity for health care technology companies working in spine, orthopedics and cancer. A group of companies are experimenting with computer vision and tiny instruments to drive into the lungs and detect cancer cells.

Gone are the days when it would have cost $2.7 billion for scientists to set out to sequence the human genome. Today, consumers can have a gene of their DNA read at a reduced cost. This would open up a new powerful tool in cancer diagnosis and research. Frequent genomic analyses can easily detect mutations associated with cancer can be diagnosed at an early stage through gene mutation in specific cells.

Therefore, 2020 will be the year for different technologies like wearables, robotic surgery and cutting-edge genomic technology other than telemedicine.

See the rest here:
Will 2020 be the Year of Telemedicine? - Medical Tech Outlook

We wish we’d written that: STAT staffers share their favorite stories of 2019 – STAT

As we look back on 2019, we at STAT find ourselves a little jealous.

There has been a lot of stellar health and science journalism this year, and below is a roundup of the stories we wish we had written.

And wed be remiss if we didnt admit the origins of this annual tradition Bloomberg Businessweek did it first, and head over there for more great reads.

advertisement

Story by Blake Ellis and Melanie Hicken, CNN InvestigatesPhotographs by Melissa Lyttle for CNN

Teaira Shorters appendix ruptured while she was in jail, serving time for minor offenses such as not wearing a seatbelt. She began to experience symptoms while in custody but her pleas for medical help were ignored for days which ultimately resulted in a life-threatening infection.

This investigation of an individual case sheds light on institutional problems in our foster care and prison system that put vulnerable populations at terrible risk. Melissa Lyttles photographs bring us directly into the life of this young woman trying to move forward. Contributed by Alissa Ambrose

By Ryan Cross, Chemical & Engineering News

When a young man in Wilsons clinical trial of a gene therapy died, in 1999, it basically shut down the field for a decade and made Wilson a pariah. C&ENs profile shows us not only how the tragedy made Wilson reassess his approach to science but also how it turned him into one of gene therapys most outspoken critics: Although he believes deeply that repairing genes can cure some of our most devastating diseases, Wilson is also outspoken about the risky approaches that some gene therapy studies are taking today. Contributed by Sharon Begley

By Ava Kofman, ProPublica

To read this piece is to see todays equivalent of a Dickensian debtors prison. Ava Kofman lays out, detail by infuriating detail, how digital technologies touted as progress are used to criminalize poverty. Supposedly, installing ankle monitors is a way to get people out of jail. But because companies charge the wearers daily fees they often cant keep up with and because their devices make it especially hard to land or hold down a job the practice ends up sweeping more people behind bars. Kofman masterfully weaves a tale of bodies controlled by private firms, of lives upended by machines that were supposed to set them free. As one young man puts it, I get in trouble for living. For being me. Contributed by Eric Boodman

By Martin Enserink, SciencePhotography by Tom Bouyer, Expedition 5300

Journalist Martin Enserink journeyed high into the Andes to write about research into the effects of chronic mountain sickness traveling, effectively, into thin air. He and photographer Tom Bouyer, whose striking photographs make this a visually arresting piece, traveled to La Rinconada, Peru, the worlds highest settlement and a gold mining town. If that activity draws to mind the wild, wild west, hang on to that thought. Enserink described La Riconada, which is north of Lake Titicaca, as Madmaxian, observing that the researchers typically retreat to their hotel rooms by 8 p.m. for safetys sake.

This forgotten part of the world is perilous for other reasons. People living in an environment with half the oxygen available to lungs at sea level can experience a host of physical ailments. These researchers would like to pave the way to therapies for chronic mountain sickness, but first need to better define what living and working at this altitude does to human bodies. Its a fascinating read. Contributed by Helen Branswell

By Ben Elgin, Bloomberg

At first, the foreboding ads flooding D.C.-area television sets didnt make much sense: Why would an advocacy group representing Americas sheriffs care whether states can import prescription drugs from Canada? Bloomberg investigated and found an answer: The pharmaceutical industry was funding the ads through an intermediary group, the Partnership for Safe Medicines. In a year already dominated by heavy-handed lobbying and advocacy surrounding prescription drug pricing, Bloomberg spotlighted one of the most brazen examples of indirect ad campaigns meant to gin up antagonism toward attempts at lowering drug prices. Contributed by Lev Facher

By Caroline Chen, ProPublica

Chens exhaustive investigation of the unregulated $2 billion stem cell industry showed how questionable marketing practices and misleading scientific claims are duping patients into paying thousands of dollars for injections of amniotic stem cells that dont work. Chens work prompted the Food and Drug Administration to ramp up its enforcement efforts. Contributed by Adam Feuerstein

By Rob Copeland and Bradley Hope, Wall Street Journal

This is the story of how Martin Shkreli, the cartoonishly disgraced biotech entrepreneur, continued to run his synonymous-with-greed drug company from federal prison. There are memorable cameos from inmates called Krispy and D-Block, fascinating details about a corporate power struggle, and an Austrian interior designer who made a regrettable investment. But the star of course is Shkreli, whose jailhouse persona lands somewhere between Jordan Belfort and Pepe the Frog. Despite lots of seemingly reasonable advice to just give it a rest, he remains convinced of his own gift for drug development and incapable of ever, for any reason, logging off. Contributed by Damian Garde

By Betsy McKay, Wall Street Journal

Our job as journalists is to notice the obvious, and this story does that brilliantly. For years, cardiovascular disease has been in decline, and it was expected to fall below cancer as the leading cause of death. In the words of Robert Anderson, chief of the CDCs mortality statistics branch, Its highly unlikely given the current trend that there will be a crossover anytime soon. In fact, the rates of heart attack and stroke mortality among people in their 40s and 50s are increasing. The story even takes a paragraph to embrace a celebrity angle, noting the deaths due to stroke of 90s icons John Singleton, who directed Boyz N the Hood and Luke Perry, who played bad boy Dylan McKay on Beverly Hills, 90210. But the story does more, explaining how heart disease patients have changed over 20 years. Once, they were men who smoked and had sky-high LDL levels. Now they are younger, more obese, and more likely to be women. The big question left behind is what society can do to put cardiovascular disease back in decline. Contributed by Matthew Herper

By Mike Hixenbaugh and Keri Blakinger, NBC News and the Houston Chronicle

In this series, reporters from NBC News and the Houston Chronicle reveal how incorrect determinations of various forms of child abuse have imprisoned relatives or separated them from children. These are incredibly complicated stories involving vulnerable children, and they show how difficult it can be to distinguish between accident and abuse. But the series reveals the ties among childrens hospitals and child welfare and law enforcement agencies and the authority conceded to doctors by the legal system. What comes across is how parents worries about a sick or injured child might just be the start of their nightmare. Contributed by Andrew Joseph

By Nellie Bowles, New York Times

Weve all heard the stories of the Silicon Valley pioneers who, after having gotten us all hopelessly addicted to our phones, now carefully limit their own childrens screen time. In this smart and provocative news analysis, reporter Nellie Bowles examines that phenomenon as well as its flip side. She tells the story of a health-tech startup called Care.Coach that employs workers in the Philippines and Latin America to operate digital avatars that live within tables and are being tested as companions for low-income seniors in the U.S. Its a telling example, she writes, of a growing class divide in how care, education, and all those services and interactions that make up our lives get delivered. As more screens appear in the lives of the poor, screens are disappearing from the lives of the rich, Bowles writes. Its an observation thats lingered with me and shaped how I, as a health-tech reporter, think about covering the growing number of health-care inventions that get delivered through screens. Contributed by Rebecca Robbins

By Anna Edney, Susan Berfield, and Evelyn Yu, Bloomberg Businessweek

Bloombergs Anna Edney has owned the generic drugs might kill you beat literally all year long, from three features over three days in January to a cover story in September to right up to the week she started her maternity leave. (Congratulations, Anna!) Pharmaceutical manufacturing and quality control is rarely the flashiest or the easiest thing to write about. But she and her colleagues showed real problems in the oversight of generic drug factories in the U.S. and overseas and illustrated the consequences lackluster oversight can have for real people. My hat is also tipped to Justin Metz, who did the simple and perfect cover photo illustration for one of Edneys stories in the Sept. 16 edition of Businessweek. Contributed by Kate Sheridan

By Sarah Zhang, The Atlantic

The Atlantics Sarah Zhang has done fantastic reporting this year on the cultural ramifications of consumer DNA testing, including this story about an Indiana fertility doctor named Donald Cline. Decades ago, Cline allegedly used his own sperm to impregnate his patients without telling them. DNA tests from 23andMe and Ancestry.com have turned up at least 50 children Cline fathered with his patients. This story told with sensitivity and gripping detail examines how those children found each other and how Clines actions have impacted their lives. Contributed by Megan Thielking

Excerpt from:
We wish we'd written that: STAT staffers share their favorite stories of 2019 - STAT

The First Cell: Dr. Azra Raza on Why the Slash-Poison-Burn Approach to Cancer Has Failed – Democracy Now!

This is a rush transcript. Copy may not be in its final form.

NERMEEN SHAIKH: Why has there been so little progress in the war on cancer? According to the director of the National Institutes of Health, Dr. Francis Collins, quote, Americans are living longer, healthier lives. Life expectancy for a baby born in the U.S. has risen from 47 years in 1900 to more than 78 years today. Among the advances that have helped to make this possible are a 70% decline in the U.S. death rate from cardiovascular disease over the past 50 years, and a drop of more than 1% annually in the cancer death rate over the past couple of decades. A drop of just over 1%? For the trillions of dollars that have been poured into cancer research, just over 1%? Today, we spend the hour with a renowned oncologist who says we should be treating the disease differently.

AMY GOODMAN: In her new book, The First Cell: And the Human Costs of Pursuing Cancer to the Last, our guest for the hour, Dr. Azra Raza, notes we spend $150 billion each year treating cancer, yet a patient with cancer is as likely to die of it today with a few exceptions as one was 50 years ago. She argues experiments and the funding for eradicating cancer look at the disease when its in its later stages, when the cancer has grown and spread. Instead, she says, the focus should be on the very first stages, the first cell, as her book is titled. She says this type of treatment would be more effective, cheaper and less toxic.

NERMEEN SHAIKH: Dr. Raza criticizes what she calls the, quote, protocol of surgery, chemotherapy and radiation the slash-poison-burn approach to treating cancer, which she says has remained largely unchanged for decades. She calls for a transformation in the orientation of cancer research, writing, quote, Little has happened in the past fifty years, and little will happen in another fifty if we insist on the same old, same old.The only way to deal with the cancer problem is to shift our focus away from exclusively developing treatments for end-stage disease and concentrate on diagnosing cancer at its inception and developing the science to prevent its further expansion. From chasing after the last cell to identifying the footprints of the first, Dr. Raza writes.

AMY GOODMAN: Well, for more, Dr. Azra Raza joins us to speak in her own words. Oncologist and professor of medicine at Columbia University, shes also the director of the MDS Center. MDS is myelodysplastic syndromes, a form of bone marrow cancer. In her book, she notes, again, that we spend $150 billion each year treating cancer, yet a patient with cancer is as likely to die of it today than one 50 years ago it is an astounding fact with a few exceptions.

Dr. Azra Raza, welcome back to Democracy Now! Its great to have you with us. How can this be? How can it be, the lack of progress that has been made in this last half-century?

DR. AZRA RAZA: Thank you for having me again, Amy. Im delighted to be here.

Since 1903, it has been well appreciated, actually, that its not cancer that kills; its the delay in treatment that kills. So, forever we have been making attempts to try and diagnose this disease early. In the last three decades, we have seen a 26% decline in cancer mortality, which is about 1% a year, as you pointed out. But thats not happened because we have developed some grand, new treatment strategy. It has happened because of two main things. One is the anti-smoking campaign, so the incidence started going down. And second is because we started using screening measures to diagnose cancers earlier and earlier.

This approach of preventive medicine, where you catch the disease early and intercept early, is what caused the drop in cardiovascular disease by 70%, because, there, the cardiologists were smarter than oncologists. They realized that if they allow a myocardial infarction, or a heart attack, to damage the heart muscle, then the only treatment would be a heart transplant, which is so draconian and so terrible. They started to diagnose not only earlier and earlier, but try and prevent the appearance by using anti-cholesterol drugs, for example. Thats a very clear case of early detection, but then even prevention of the disease. And 70% decline in mortality. Why arent we doing the same in cancer?

NERMEEN SHAIKH: So what is the answer to that?

DR. AZRA RAZA: Well, the answer is that we have been trying to do it. And the screening measures that were put in place, like mammography, colonoscopy, PSA testing, Pap smears, theyre the ones that caused the decline by 26% mainly, in addition to anti-smoking campaigns. But those measures were put in 50 years ago. Imagine, in this day and age of technology, we are still putting a tube into someones gut and looking to find cancer. That is primitive. Thats paleolithic for today.

AMY GOODMAN: And the alternative is?

DR. AZRA RAZA: The alternative is that we have milked these technologies as much as we could . They have yielded the 26% decline in mortality. Theyre not going anywhere else. We need to invest in developing technology based on current imaging, scanning devices, detection of biomarkers, for example, from blood, sweat, tears, saliva, urine, stool samples, and find the earliest footprint of cancer and see how we can intervene. And this is a strategy that is not limited to just cancer, Amy. This is a strategy that is going to apply to every single chronic disease in the coming years.

AMY GOODMAN: So, what has prevented that from happening?

DR. AZRA RAZA: I wish there was a very neat kind of answer about this, but its something like this. You take a frog and put it in cold water and start heating it slowly; nothing is going to happen. On the other hand, you throw a frog into boiling water, it will jump out. But if you heat it slowly, the frog dies without jumping out, because it slowly gets used to it. This is an apocryphal story, by the way; scientifically, its incorrect. Just a warning. As a purist, I have to add that. But the analogy is true, that things have happened so slowly that we keep getting desensitized to the next step. One thing is that there is so much hyperbole around cancer treatment. If a few months of survival are added by a drug, it is welcomed as a game changer. Let me

AMY GOODMAN: At the end of life.

DR. AZRA RAZA: Yes, exactly. Let me give you just a few statistics. I want to be very clear that using the slash-poison-burn approach, we are curing 68% of cancers that are diagnosed today. We are curing them. Thirty-two percent that present with advanced disease, their outcome is the same exact outcome that it was 50 years ago. The 68% we are curing, why are we still using these Stone Age treatments? You know how terrible it is to get chemotherapy and radiation therapy.

AMY GOODMAN: Explain how it works.

NERMEEN SHAIKH: Explain what yeah, yeah. What happens?

DR. AZRA RAZA: The first rule of medicine is first, do no harm. In fact, when a patient is diagnosed with cancer, its a silent killer. Thats the problem. It can reach stage IV disease without producing symptoms. So, somebody comes to us I recently saw a 42-year-old young man who has just finished a game of tennis and come to see me, and suddenly, because he was exhausted and feeling so tired, and now I diagnosing him acute myeloid leukemia. I look at this toned and tanned young man, and the first thought that comes to me is about what we are going to do to him with the chemotherapy we are going to give him.

It is unconscionable that in 2019 I am still going to give this young man the same combination of two drugs that we popularly known as 7+3, that I was giving in 1977, when I arrived in this country. I feel ashamed of myself, having to repeat the same side effects, that you are going to lose all your hair, throw your guts out, and your counts are going to tank. Your blood counts will go down to essentially zero for weeks on end, where you are going to be susceptible to all kinds of terrible infections. You will be in the hospital suffering with shivering night sweats and fevers and all kinds of aches and pains and constitutional symptoms. And then there is a chance that a percentage of those patients will improve. So, this is what we do with just chemotherapy alone.

AMY GOODMAN: Were going to break and then come back to this discussion and also hear about your personal story with your own husband, also a renowned cancer doctor, who died of the very disease that he was studying, and hear the stories of your patients. Were talking to the renowned cancer doctor, the oncologist, the professor of medicine at Columbia University, Dr. Azra Raza. She has a new book. It is called The First Cell: And the Human Costs of Pursuing Cancer to the Last. Stay with us.

[break]

AMY GOODMAN: Nocturnes, Op. 27, No. 2, in D-flat major, _lento sostenuto _, performed by Abbey Simon. Abbey Simon passed away December 18th at the age of 99. This is Democracy Now!, democracynow.org, The War and Peace Report. Im Amy Goodman, with Nermeen Shaikh, as we spend the hour with Dr. Azra Raza, a renowned oncologist, professor of medicine at Columbia University, where shes also the director of the MDS Center a form of bone marrow cancer her new book, just out, titled The First Cell: And the Human Costs of Pursuing Cancer to the Last.

NERMEEN SHAIKH: So, Azra Apa, Dr. Raza, we were talking earlier, in the first part of the program, about the slash-poison-burn approach to treating cancer, which youve been very critical of, which involves surgery, chemotherapy and radiation. You say that this has been responsible for curing 68% of cancers. So a couple of questions: Would it have been possible, even though this is counterfactual, to cure such a high number of cancers using alternative methods? And, two, what kinds of advances have been made in how chemotherapy is administered and how the effects of that, of changes in the way that its administered, have had on patients?

DR. AZRA RAZA: Both very good questions. So, the first question you asked is what could have been really done. And what have we been aiming at doing? So, sure, we started by using these blunt approaches. Its literally like taking a baseball bat to hit a dog to get rid of its fleas. Thats how bad this kind of treatment is. But we had to do it because we had no alternatives. In the meantime, we invested billions of dollars in trying to study the biology of cancer, hoping that we will identify some intricate signaling pathway, some genetic defect, that is going to allow us to target it specifically. And this did happen in two diseases. In chronic myeloid leukemia, we developed a targeted therapy, because theres one gene had gone wrong, and one magic bullet could target it and cure the disease. Now we

AMY GOODMAN: And again, just to note, youre one of the worlds leading authorities on AML, this kind of cancer.

DR. AZRA RAZA: On this, Im talking about chronic myeloid leukemia. But yes, youre absolutely right: I am an expert in myeloid diseases. And CML, chronic myeloid leukemia, is something Ive treated for decades.

Now, this was a huge advancement that happened in the early 2000s, that we could now use a targeted therapy which is not chemotherapy, which only goes and attacks the abnormal cell which is expressing this protein. While it helped patients, its also put the field behind by 20, 30 years. Why? Because we felt that this now establishes a paradigm. Every cancer will be caused by one genetic defect, for which we just have to develop one drug. So, one gene, one targeted therapy. Everybody and their grandmother has been trying to find the one gene for pancreatic cancer, the one gene for acute myeloid leukemia. It turns out that, unfortunately, for all other cancers, most of them, really, there are too many genes that are mutated simultaneously. And so the targeted therapies weve developed, even for those multiple proteins or one protein that is dominant, it turns out it works for a little bit.

So, let me give you some statistics again. Ninety-five percent 95% of the new drugs that we are bringing, the experimental drugs we bring to the bedside, 95% of them fail. And to bring one of those drugs to the bedside is a billion dollars almost. So imagine how much we are losing. Five percent that succeed should have failed, in my opinion. Why? Because theyre only prolonging survival by a few months. So, for example, theres a drug that extends the life of a pancreatic cancer patient by 12 days, at the cost of $26,000 a year and not every pancreatic cancer patient, just a fraction. So think of the financial toxicity we are causing to these patients now. For very little prolongation and survival, we are financially ruining 42% of cancer patients diagnosed, newly diagnosed with cancer today, by year two. They lose every penny of their life savings.

AMY GOODMAN: Speaking of which, before we go on to the incredible stories you describe in The First Cell, your thoughts on Medicare for All? I have spent a lot of time accompanying family and friends, for example, at Sloan Kettering, who are dealing with cancer. The astounding devastation of peoples, aside from lives, financial destruction. Medicare for All, what would that mean for cancer patients?

DR. AZRA RAZA: Amy, while Im not an expert on these issues, I have common sense. And one thing I can say is that the current situation is completely untenable. We are on the verge of a collapse with the healthcare system. We cant continue it the way it is going. And, to me, the only solution seems to be but again, its not speaking as an expert is to have Medicare available for everybody. I think thats the only compassionate and humane solution.

NERMEEN SHAIKH: Well, Azra Apa, I want to ask you about another incident that you mention in the book, before going on to the longer case histories of patients in their own words as well as in the words of their family members. I mean, the cost of cancer treatment, as you say, in the U.S. is exorbitant, and many families have been driven to financial ruin. But what about in places where cancer treatment is so prohibitive that it entirely deprives patients of access? You talk about arriving in Karachi and meeting in Karachi, Pakistan, which is where youre from and talking to Zaineb, a cancer patient about whom you write, quote, How does one go in and talk to a thirty-five-year-old woman for whom dying from leukemia is only her second-biggest problem? Could you talk about that, access to cancer treatment and who the people are who are entirely deprived of any treatment at all?

DR. AZRA RAZA: This is a subject which is very close to my heart, Nermeen, because, obviously, I am from that part of the world. The entire world looks to America for leadership. What kind of leadership are we providing? A leadership where we are developing drugs or cellular therapies now that will cost over a million dollars per patient, for helping very few patients. These are rarified cases for whom this kind of treatment will work. As you said, what about the 20 million new cancer patients being diagnosed universally all around the world? We just dont have a compassionate solution for them. Why arent we thinking not just of those even in America, in the remote areas and many, many places where healthcare is becoming harder and harder to access just because hospitals are closing down from financial ruin themselves? Why arent we thinking of them? Why arent we thinking beyond American borders, for humanity as a whole?

And when you think about that solution, I mean, you dont have to be a genius. No, no, look, the only thing that works in cancer if you have someone who gets diagnosed with cancer, the first thing youll say is, Was it advanced, or was it caught early? No, early, so theres hope. We know that early detection helps, right? Why are we investing money in chasing down the last cell, in which with drugs which have a 95% failure rate today? Because the pre-clinical testing platforms we are using are so artificial, and thats why we are bringing up drugs that are not going to be successful.

Instead of doing that, why arent we simply improving on the techniques we know work? What worked? Early detection. How do you do early detection? Well, mammography, colonoscopy, Pap smear, etc., has been reached to their maximal limits. Lets develop the new technology so that from one drop of blood, you can put it on a little chip, which can be read by a cellphone, which costs $180 for 12 cancers to be diagnosed early. This is something that was just announced by a Japanese company. So these are things not that are a pie in the sky. These are things that are happening now. Except how fast will it happen to reach the rest of the world? It depends on the amount of resources we invest.

AMY GOODMAN: Were talking to Dr. Azra Raza. Her book is called The First Cell. She is a leading oncologist in the world. She is a Pakistani American, a Muslim, a woman. You talk about your own story in the book, the story of your husband, also a leading oncologist. Dr. Harvey Preisler died of lymphoma in 2002. You write, Cancer is what I had been treating for two decades, yet until I shared a bed with a cancer patient, I had no idea how unbearably painful a disease could be. Lets first turn to your daughter, to Sheherzad Raza Preisler, speaking at the memorial service for her father, your husband, about his cancer diagnosis and the effect his condition had on the family. The 15th Harvey Preisler Memorial Symposium was held in New York in 2017.

SHEHERZAD RAZA PREISLER: What a cruel twist of irony it was that as he was directing the Rush University Cancer Center in Chicago, he was cut down in the prime of his life by the very disease he had dedicated his life to cure. I was only 3 when he was diagnosed and 8 when he died. My parents took great pains to never mention the C-word in my earshot, and yet most of my memories of Dad are related, at least in part, to the presence of this nameless other in our lives. And even though I was too young to know what was going on at any tangible level, I had some sort of instinctual knowledge that something was terribly wrong. I could sense my mother struggle as she was navigating through stages of optimism, pain, dread, despondency and, eventually, hopelessness, as my dad underwent a seemingly endless stream of experimental treatments. These stages are what most cancer patients and their caregivers and families experience.

AMY GOODMAN: That was Sheherzad Raza Preisler, the daughter of our guest today, Dr. Azra Raza, oncologist, professor of medicine at Columbia University, talking about her dad, who died of one of the cancers that he specialized in. He was head, Dr. Harvey Preisler, of the Rush Cancer Institute in Chicago. Tell us, as you begin your book, what happened to your husband, what happened to Harvey.

DR. AZRA RAZA: First of all, Amy, when I started writing this book, I had no intention of putting Harvey into the book. I was writing about my other patients. But when I wrote about them in such detail and with such painful granularity, I felt it would be dishonest if I held back my own story. And so, at that point, I decided to add. And once I decided that, this story became like a red line running throughout the whole book, because then I couldnt escape, at every level, where Harvey came in.

Ill give you just one example. I mean, he was the head of the cancer center. He gets the very disease hes trying to cure. And now he dies, after a very, very exceedingly painful almost five-years battle. Sheherzad was 8 years old when he died. And a couple of weeks after he died, she developed a terrible cold and flu and was pretty sick for a few days and then started getting better. And one morning I was sitting in the living room reading, and she suddenly comes out crying inconsolably. I was sure that shes had a relapse and is much worse. But when I was able to calm her down, this is what she said, Amy. She said, Actually, I feel perfectly fine now. But I know what it feels like to be so sick and how good it is to get better. And my dad never got better. Which brought on a second fit of crying. So, an 8-year-old, at a visceral level, is able to experience the kind of anguish that cancer patients suffer.

Being a cancer widow, did it make me into a different kind of doctor? No. But my perceptions changed entirely. You know, Marcel Proust has said that real voyage does not lie in finding new landscapes, but in having new eyes. And so it became a voyage of discovery for me, in having new eyes.

AMY GOODMAN: And that issue of prevention, of catching early, what happened in your husbands case? Who I mean, he was head of a major cancer institute.

DR. AZRA RAZA: The same thing that happens to all other cancer patients: He was diagnosed way too late. And actually, the lymphoma didnt kill him. The treatment we gave killed him. The chemotherapy we gave destroyed his immune system, so that he died eventually of sepsis. He didnt die of the lymphoma.

Theres a family friend of ours who once told my younger brother he said, Abbas, if the sun rose suddenly from the west one day, the entire world will stop and stare at it. But there are some people who watch the sun rise from the east every day, and they wonder why. And he said, Those are the only kinds of people who can make a difference in the world.

And thats the kind of person Harvey was. He was an extremely thoughtful person with a great curiosity about life in general, but specifically scientific issues. Yet what I learned from taking care of him and sharing a bed with him as a cancer patient was a deeply humbling acceptance of his condition. Basically, his attitude was I am a man, and a man is responsible for himself, even though I know that this is not going anywhere.

AMY GOODMAN: You write at the beginning of your book, in your introduction, From Last to First, talking about that idea of the first cell, I have learned new things about what I thought I already knew: like the difference between illness and disease; between what it means to cure and to heal; between what it means to feel no pain and to feel well. Can you elaborate on this?

DR. AZRA RAZA: I mean, really, the whole concept of this is can be expressed better in poetry. So, if you dont mind, I will recite a short piece by Emily Dickinson:

I measure every Grief I meetWith analytic eyes I wonder if It feels like Mine Or has an Different size.

I wonder if They bore it long Or did it just begin I cannot find the Date of Mine Its been so long a pain

I wonder if it hurts to live And if They have to try And whether could They choose between They would not rather die.

NERMEEN SHAIKH: Well, I want to ask about another patient who you profile in the book, who is in fact subjected to a particularly brutal form of this slash, burn and poison. And thats Andrew Slootsky, who died in 2017 at the age of only 23. The book includes a testimonial from his mother, Alena, who criticizes the attitude of the oncologists who treated him, but says she would have wanted Andrew to undergo any treatment that might have extended his life. So, could you talk about Andrew was also one of the best friends of your daughter, Sheher. Could you talk about the treatment that he was subjected to? What happened? And also your decision one of the most remarkable things about this book is not just, of course, your expertise in cancer and cancer treatment, but also these stories that you weave into the narrative, of patients youve treated, of patients families who have witnessed their family members being treated in this way and ultimately dying. And this is Alena, Andrews mother, saying that she would have Andrew go through all of this treatment, even if it meant the slightest possibility of extending his life.

DR. AZRA RAZA: Yes. Nermeen, actually, Andrew is the impetus for me to write this book. In 2016, when he gets diagnosed with cancer, he has weakness in his arm. He goes to the emergency room. They do an MRI. They find an inoperable brain tumor, nine centimeters long. They couldnt remove it. From the day one, every oncologist that treated Andrew knew that his chance of survival is 0.00, beyond what is to be expected. No matter what we give him, he was going to die. We all knew it.

This boy, when he opened his eyes, coming out of anesthesia, he turned to his mother, and he said, Mom, dont worry. Just call Azra. Shes on the cutting edge. She will find the best treatment and cure for me. And Alena called me. This young man, whos been in and out of my house since hes 15 years old, because hes best friends with my daughter. I felt so ashamed of myself that there is nothing I can offer this poor boy, and the fact that we are failing the Andrews and the Zainebs and the Harveys and the Omars of this world so spectacularly, and instead of feeling embarrassed, we go around pumping our chest, claiming that we are curing 68% patients. Sixty-eight percent of what? again and again I ask.

And what we do to Andrew in the next 16 months, even though we knew his chance of survival is zero, is we give him chemotherapy, radiation therapy, surgery after surgery, more chemo, more radiation, more immune therapy. And he suffered the side effects of every one of them, without benefiting from anything.

But then, the converse is also a problem. Lets say we didnt treat him and let the brain tumor take over. That death from advanced cancer is just horrendous. So, basically, the treatment, what are the choices we offered Andrew at this point? Either you die of your cancer or die of the treatment, but you are going to die. And the question I ask myself is Why? Why was Andrew diagnosed when his cancer was nine centimeters long? We know that cancer is a silent killer. We know no age is immune to getting cancer. At 22, this boy got cancer.

AMY GOODMAN: And specifically in his case, how could it have been detected earlier? Talk about the tests that you think need to be developed, and are actually already there but somehow missed him?

DR. AZRA RAZA: So, Amy, I am calling for a complete paradigm shift. What Im saying is that even the screening measures that we are using annually, for example, doing a mammogram is too late for some people. What we need to do is consider the human body as a machine and continuously learn to monitor it for the detection of diseases, whether its Alzheimers or cancer or diabetes. We need to catch all of these diseases early and try to nip them in the bud. This is what Im asking for. So, what has to be done for that? Children get cancer. Not that many, but certainly they do. And again, we treat them with these draconian measures, and they end up with developmental issues, fertility issues, all kinds of problems. So, what Im asking for is we need to develop those markers that can identify cancer at its inception. It is doable. It is possible.

A lot of research is going on, in this country and elsewhere also, to find footprints of cancer, for example Ill give you a couple of examples. When cancer starts, it divides fast its cells divide faster than normal cells, which means it needs more nutrition, so it starts making new blood vessels. As soon as that happens, the area becomes hot because of new blood going into that one area. This hot area can be detected. People are developing bed sheets and mattresses where you can go to sleep and you are overnight youre being scanned for hot areas. Lets say a hot area is detected in my pancreas one day. It doesnt mean the next morning I should have an open abdominal surgery and eviscerated and removal of all my no, it means that now theres something abnormal which needs to be monitored. I am now considered someone at high risk, so I should be monitored for other biomarkers. These cells which are developing in my pancreas will be shedding their proteins into the bloodstream. If theyre not shedding, you know one thing we can do? We can yell at them. How do you yell at them? You use sound waves, literally, ultrasound, to hit them. And they start shivering, and they start shedding their proteins into the blood. And we get the blood and detect the biomarker.

AMY GOODMAN: Is there a cancer-industrial complex thats preventing this kind of research and development of the preventive and the early-detection approaches to cancer?

DR. AZRA RAZA: Absolutely not, Amy. In fact, my contention and my conceit is that if heres an industry that is investing in an enterprise that has a 95% chance of failure, but they keep investing billions of dollars, because if one of their drugs makes it, if it improves survival by 10 seconds more than two months

AMY GOODMAN: So youre saying there is a cancer-industrial complex that is

DR. AZRA RAZA: But theyre not preventing it.

AMY GOODMAN: Right.

DR. AZRA RAZA: They just dont know whats a better thing to do. So, what Im saying is, we just set a new goal, and we financially incentivize the goal, then all these people will turn around and come to the first cell instead of going after the last cell.

AMY GOODMAN: What about Vice President Bidens moonshot challenge around curing cancer?

DR. AZRA RAZA: I was one of the fortunate people to meet Vice President Biden in his across his dining room table for the cancer moonshot and had a very wonderful discussion with Vice President Biden. His heart is in the right place. And there is a certain fraction of that billion dollars, the money that he has allocated for cancer research, is definitely towards prevention and early detection of cancer. But its not enough. That kind of vision is what we really need. But we need Im not saying all current research should stop. Nobody should misquote or mishear me. I am saying we have correct patients. Of course we have to worry about them, and we have to keep developing better treatments and better understanding of biology. But I think at least half of the resources, and all the resources going into these failing clinical trials, these billions and billions of dollars, can be redirected for future patients to try and detect the disease early a solution which will be applicable universally.

AMY GOODMAN: We have to break. Then were coming back to our guest, Dr. Azra Raza, oncologist, professor of medicine at Columbia University, where she directs the MDS Center, a form of bone marrow cancer. Her new book is out. Its called The First Cell: And the Human Costs of Pursuing Cancer to the Last. Stay with us.

[break]

AMY GOODMAN: New York by St. Vincent. The photos in the video were compiled by Kat Slootsky for her brother Andrew, one of the patients profiled in Dr. Azra Razas book, The First Cell: And the Human Costs of Pursuing Cancer to the Last. He died when he was 23 years old, of a particularly aggressive form of brain cancer. Im Amy Goodman, with Nermeen Shaikh. Were spending the hour with Dr. Azra Raza, the renowned oncologist, professor of medicine at Columbia University. Nermeen?

NERMEEN SHAIKH: So, Azra Apa, I want to ask you about one of the criticisms that your book has come under. In The New York Times, Dr. Henry Marsh praises the book but says youre too optimistic about the solutions you propose. Marsh writes, quote, Her diagnosis of the ills from which cancer treatment suffers strikes me as accurate, but her solutions seem infused with the same unrealistic optimism she identifies as the cause of so much suffering. This is Dr. Henry Marsh writing in The New York Times. Are you guilty of too much optimism?

DR. AZRA RAZA: No. Thats the short answer. But, Nermeen, you know, I started my career in oncology basically in 1977. I was 24 years old, fresh out of med school. I had come here. I started working at Roswell Park Cancer Institute, because I was going to cure cancer very quickly, I thought. And within seven, eight years, it was very clear to me, the disease that I had invested all my energies in, acute myeloid leukemia, that in my lifetime this disease will not be cured. It is so complicated.

So, at that point, I turned my attention toward studying an earlier form of the disease, because many of my patients who came with acute leukemia gave a history of having had some low blood counts and being anemic for a few months before it developed to leukemia. So I said to myself, Why not catch this disease earlier, the pre-leukemia phase, and intercept then and not let it become this end-stage monstrosity?

So I started collecting this is where being an immigrant helped me, Amy, because had I gone to school in this country, if I had started to study acute leukemia, my next step would have been to make a mouse model of this disease, which are very artificial and which just for drug development, at least. Theyre very good models for studying biology, but not for drug development. But because I was an immigrant, I simply started saying, Oh, I have to study cancer, so let me save these cells. And I started banking cells on my patients. Today I have a tissue repository which has 60,000 samples from thousands of cancer patients, followed longitudinally, well annotated, with all their clinical and pathologic data in the computerized forms. And this is a very precious resource, one of a kind. Not one single cell comes from another physician. I still do the bone marrows with my own hands, which Im going to do in the next hour, when I get to my clinic.

But the idea I had was that earlier detection will help. That was many years ago. Wheres my solution? So, the question you ask me and the criticism that Dr. Marsh is giving can be applied to me, yes, that 35 years ago I felt that detecting the disease early, pre-leukemia, would help me. It hasnt helped me, because pre-leukemia itself is a very malignant disease and can kill.

So then I realized that by using these samples of acute leukemia, working my way back to pre-leukemia, I can then ask the question: Why did some people get pre-leukemia, to begin with? Why did they get MDS? And once we can discover that, by using these tissue samples and the latest technology of proteomics, genomics, transcriptomics, metabolomics, panomics, we will find the same kind of thing you have, say, for breast cancer, the BRCA gene. Can we find something thats making people susceptible because of their inherited DNA?

AMY GOODMAN: What about targeted immunotherapy? People think that it has gone so far to help cancer patients. What is it? And what do you see are the prospects for it?

DR. AZRA RAZA: The answer to that is, first, you have to understand what is the cell therapy. So, there are many, many different forms of immune therapies, fourteen, fifteen different kinds. But the ones that are receiving all the attention are basically two types. Checkpoint inhibitors are drugs which cancer cells so, every cell expresses proteins that either say, Eat me, or Dont eat me. Cancer cells learn to express only proteins that say, Dont eat me. Dont eat me. Dont. So, this is how they deflect the immune system.

AMY GOODMAN: We have less than a minute.

DR. AZRA RAZA: And so, we tried drugs to do that. We succeeded. The response doesnt last. The other form of therapy is cell therapies that we use. Immune therapy using cells T cells, for example cannot distinguish between a normal cell and a cancer cell in the organ. All we can do is we can activate these T cells and say, Go kill the whole organ, and cancer will die with it. So the only cells we have succeeded in killing, or the only organ we have succeeded in killing, so far, is B cells, which are a kind of lymphoid cells in the body.

AMY GOODMAN: Ten seconds.

DR. AZRA RAZA: We kill them, and then we replace B cell function by giving immunoglobulins for the rest of their time. We cant do that for the liver. We cant kill the liver and expect to replace it. Thats why immune therapy using cells is not going to work.

AMY GOODMAN: Well, were going to have to leave it there, but people can pick up the book and take it from there. Dr. Azra Raza, oncologist, professor of medicine at Columbia University, heads up the MDS Center, a form of bone marrow cancer. Her new book, The First Cell: And the Human Costs of Pursuing Cancer to the Last. Im Amy Goodman, with Nermeen Shaikh.

Go here to read the rest:
The First Cell: Dr. Azra Raza on Why the Slash-Poison-Burn Approach to Cancer Has Failed - Democracy Now!

Christmas In The City Thrives As Founder Battles ALS – WBUR

It's a Here & Now tradition that began in 2015 when the show first aired a holiday story about Boston's Christmas in the City, the world's biggest party for homeless children.

Since then, we've been sharing those kids' joy with our listeners, just as Christmas in the City has continued throwing its massive holiday party.

The event, founded as a 100% volunteer nonprofit by Jake Kennedy and his wife Sparky Kennedy, kicked off in 1989.

As Jake Kennedy explains, it started when their daughter was 2 years old.

"She got too many presents so she had no idea what Christmas meant and you know, we wanted to show her the other side of life, he says.

The first time they held Christmas in the City, the event was small, with only a few hundred kids at their family business, Kennedy Brothers Physical Therapy. Over time, the event took off.

By 2015, the number was around 4,000 people, and this year, about 6,000 kids were in attendance. The event is packed with presents, food, carnival rides, face painting, and of course, a visit from Santa Claus.

Jake told us in 2015 the event made him emotional.

"You don't understand people who have to wait in line, that no promises are kept, they don't know what school they're going to, they don't know where they're going to live, they don't know where the next meal is coming from, he says. What it means to be treated like princes and princesses."

"I wouldn't miss it," he continued. "I wouldn't miss it."

Speaking to him last week, the enthusiasm and the spirit are the same though his voice is lower, his speech slightly slurred. Because the man who's dedicated his life to fighting others' battles is now fighting his own.

He's been diagnosed with ALS also known as Lou Gehrig's disease an insidious disease that leaves the mind intact but ravages the body. Jake's father died of ALS, as did Jake's younger brother he called Squirrel. Another brother, Ratt, also has the disease.

Sparky says the diagnosis didn't come as a surprise. In fact, when the first symptoms appeared, she said she knew immediately.

"Jake was tested for the gene about five years ago. October 11 was when it was confirmed it was the ALS, she says. I noticed the symptom of stuttering speech the very last week in August, actually."

Jake and Sparky said they had a brief reprieve when Jake tested positive for Lyme disease, which could have produced that symptom. But in the end, it turned out that they were facing ALS.

Despite the diagnosis and the family history, Jake is anything but defeated. "I'm very optimistic about a cure," he says.

This is a time like no other time in research. And Dr. Brown, Zack, they are so optimistic, he adds.

Hes referring to his son Zack, a post-doctoral researcher at UMass Medical School in Worcester, Massachusetts, and Dr. Robert Brown, director of Neurotherapeutics at the University of Massachusetts.

The two are working together on ALS research.

"Approaches now for treating familial disease are very powerful in terms of inactivating the genes that cause this to run in families," Brown says. "So there's more hope for familial ALS then there's ever been."

Brown says it's also exciting that the lessons from the genetic forms of ALS also apply to the other 90% which are not familial. Zack adds that there are clinical trials that may benefit his father.

For Zack, family history was among the factors that drew him into ALS research.

"It's very motivating to know that your work will go into helping your family for sure," he says, growing emotional. "There are ups and downs, it gets heavy, but nothing good comes easy."

Zack says he's impressed with how his family, particularly his mother, are handling the new reality scheduling doctor's appointments, dealing with insurance and remaining positive.

Sparky says she's just taking it one step at a time. "Putting one front in front of the other is how I'm doing it right now, she says.

In some ways, Jake says Christmas in the City has given him the perspective he needs to deal with battling ALS.

"Because of the Christmas in the City, I feel how unjust society is, he says, so I think other people are far worse off than I am, right?"

"We talked about this before you were diagnosed. If I died tomorrow, I would have had the most blessed life ever, Zack says. And life's not fair."

"And so you just have to count your blessings and take it day by day, Zack continues, his voice trembling. And I think you'd agree that we've had such a blessed life. And it's very easy to forget that and it's easy to get angry.

Yet Jake calls his 65 years on the Earth a total blessing. He mentions his four children and their spouses, who have brought so much into his and Sparky's lives.

Jakes wish moving forward is for people to reach out and change society forever. So many families are socially and economically disparaged and I want that to end."

"We want research money and we want to find a cure, Sparky adds, and we're going to remain hopeful."

When the topic of testing is raised, Zack again is emotional. He says he was tested and is negative meaning he does not carry the ALS gene. The couple has a new baby.

"It's the most wonderful blessing," he says in a whisper. "But it's hard thinking about your siblings."

Click here for the Jake Kennedy ALS Fund.

See the article here:
Christmas In The City Thrives As Founder Battles ALS - WBUR

Once winded walking up stairs, Omaha man is back in gym thanks to new cystic fibrosis therapy – Omaha World-Herald

Nicholas Bell arrived at the gym on a sunny December afternoon, climbed aboard a treadmill and jogged, occasionally slowing for a breather.

His run done, he headed for the weights. Its a routine hes been trying to fit in three or four days a week.

But until a few months ago, Bell, 36, had largely stopped working out. Just walking up the stairs of his Benson-area home left him winded.

Then in late September, he started a new therapy for cystic fibrosis, the rare, progressive disease he was diagnosed with at 6 months old.

The treatment, a three-drug combo called Trikafta, has been hailed as the first to show dramatic improvement in lung function in most people with the disease. While not a cure, its expected to benefit 90% of patients, including those who have the most common mutation in the cystic fibrosis gene, called CFTR, which was discovered 30 years ago. The Food and Drug Administration approved the therapy in late October for patients 12 and older.

Now that hes back to working out, Bell realized it wasnt motivation he lacked, but the ability to do the work.

Its exciting I can work out and still have energy to walk the dog and go be with my friends, he said of his recent turnaround.

Until a few months ago, Nicholas Bell, 36, had largely stopped working out. Just walking up the stairs of his Benson-area home left him winded. Then in late September, he started a new therapy for cystic fibrosis.

Cystic fibrosis affects an estimated 30,000 people in the United States and 70,000 worldwide. It causes thick mucus to build up in organs, damaging patients lungs and digestive systems.

In clinical trials, the drug boosted lung capacity an average of 14%, said Dr. Peter J. Jim Murphy, program director of the Adult Cystic Fibrosis Program at the University of Nebraska Medical Center/Nebraska Medicine. That program and the Pediatric Cystic Fibrosis Center at Childrens Hospital & Medical Center form the Nebraska Regional Cystic Fibrosis Center, which treats more than 300 patients. One Childrens patient participated in clinical trials of the drug.

We face, every day, steady decline in lung function in virtually all of our patients, he said. In one fell swoop, youve turned that around. Its whoops and hollers for the cystic fibrosis team, and has driven quite a few patients to tears of joy.

And while most attention has focused on the initial boost the drug provides in lung function, Murphy said, doctors also are hoping it will decrease the rate of decline in that function by as much or more as earlier drugs. Long-term results, of course, are not yet in.

All adults face a decrease in function after age 30, he said, but its typically not enough to cause problems. Cystic fibrosis patients, however, lose lung function about four times faster on average than the typical person.

They very quickly cut into the lung function they need to live their daily lives, not just to run marathons, he said.

Get the latest health headlines and inspiring stories straight to your inbox.

Bell has had his own ups and downs with the disease, including a near-death episode in 2016 that prompted his doctor to suggest it was time to consider a lung transplant. He avoided that by focusing on his health, and saw some significant improvements. But he became really ill in mid-2018 after catching a bug.

He got Trikafta a bit before the FDA approved it through a program that offered the therapy to people like himself who at the time had low lung function.

By the two-month mark, his lung function had increased by 19%, the biggest gain hes ever seen. Hes been using that improvement as a springboard to bolster his physical fitness. Hes been working to push his mile time below 10 minutes, and he plans to train for a cystic fibrosis extreme hike in Colorado next fall. He hasnt experienced any notable side effects, although hes seen posts in online groups from some who have.

With his improved energy level, hes also back to doing more writing. A teaching artist with the Nebraska Writers Collective, he coordinates a program that sends instructors into prisons to teach creative writing and also helps teach a slam poetry class at Omahas Central High School. He and his wife, Kristin, celebrated their third anniversary earlier this month.

Im happy to be where Im at now, he said.

Hes also keeping on top of his cystic fibrosis treatment routine. That includes donning a percussion vest that breaks up mucus in his lungs three times a day for between 30 and 45 minutes. He also uses a nebulizer, a device that turns liquid medications into a mist he inhales.

He encourages other people with cystic fibrosis to do the same. A clinical trial is being launched to determine whether patients can pare back their regimens, but those results arent in. He said his fear is that younger patients will decide the disease is cured and drop their treatments.

I think its important for us right now to just stick to our regimen, he said.

Nicholas Bell said his fear is that younger patients will decide that with the new therapy, cystic fibrosis is cured and drop their treatments. I think its important for us right now to just stick to our regimen, he said.

Dr. Heather Thomas, director of Childrens cystic fibrosis center, is eager for results of other studies, those testing the drug in children ages 6 to 11.

Kids stand to benefit even more than adults if the therapy can slow the diseases progression.

The earlier we can get kids on, the better, she said. So far, theres no indication the therapy wont work in younger children.

Older pediatric patients who got the drug also experienced fewer pulmonary exacerbations, meaning they didnt get sick as often. That would be a huge win for families, Thomas said.

Research also is needed to help the 10% of patients whose disease is caused by different mutations. The Cystic Fibrosis Foundation launched a $500 million campaign in late October intended to accelerate efforts to cure the disease.

Another concern raised after the drugs approval was whether patients would have access to it, given its price tag of $311,000 a year.

But Murphy said patients have been able to get the drug through insurance and patient assistance programs, typically with a relatively small co-pay. A small minority have been denied, but those denials have been reversed on appeal.

Its dramatic, life-changing and, we believe, life-prolonging, he said. Its important to know that its not a cure, and that our job as cystic fibrosis providers is still going to be here. But its going to be different.

Bell, the poet, has his own word for it.

Its freeing, he said. It definitely has been very freeing.

This report includes material from the Washington Post.

Matthew Eledge and husband Elliot Doughertyplan to explain her out-of-the-ordinary birth to their daughter in terms she can understand: that her grandmother furnished the garden where she grew, and that her aunt, Lea Yribe, generously supplied the seeds.

One pothole did a passenger a favor when the ambulance he was in struck it, according to first responders. Gretna firefighters were taking a man suffering chest pain and a high heart rate to the hospital. While en route to Lakeside Hospital, the ambulance hit a pothole. The jolt returned the patients heart rate to normal.

Thought to be brain dead, doctors took former Creighton Bluejays play-by-play announcer T. Scott Marr off life support. Before his family settled on a funeral home, they decided to see their dad one more time. When they got there, he was awake and speaking.

Karla Perez was 22 weeks pregnant when she suffered a catastrophic brain bleed and was declared brain dead. Her unborn child was alive, but wouldn't survive delivery. So family and doctors kept her on life support.Angel was born eight weeks later.

Darnisha Ladd never imagined Snapchat would help save her life after she suffered a stroke. But needing a precise timeline of events, doctors and family relied on a post on the phone app and were able to give her a needed medication in time.

Lindsey and Derek Teten's triplets are one in a million. Literally. The Nebraska City couple's three daughters, born in late June 2017, are identical and were conceived without fertility treatments. The girls were the second set of spontaneous triplets born at Methodist Women's Hospital. The first set, also girls, was born in 2015.

Doctors diagnosed the paralysis that was creeping up Justin Chenier's legs as Guillain-Barre syndrome. It would become so serious that the Omaha man would nearly lose consciousness while screaming because of the pain. The syndrome was triggered by West Nile virus.

Kenze Messman's been diagnosed with several chronic illnesses. Sometimes her heart rate climbs, seizures send her to the floor and migraines leave her in the dark. And one of the ailments causes the 17-year-old to have allergic reactions to almost everything.

The skin on Sharan Bryson's leg was black from lack of circulation. She felt nothing but a sharp, stabbing pain. The leg was dead, and her best option was amputation. Bryson bounced back and put her hard work to the test by running a 5K.

Chase Tiemann has had numerous surgeries in his young life, including the amputation of his left arm. The Omaha boy has a condition that causes tumors sometimes benign, sometimes cancerous to form on his body. To boost his spirits after amputation, the Papillion Fire Department named Chase an honorary firefighter.

Wesley Woods battled heart disease for 20 years. He'd racked up nine heart attacks, multiple surgeries and one heart transplant. He was tired of hospitals. Tired of chest pain. Tired of feeling tired. Woods was lucky he received a second transplant.

Amber Kudrna wasn't sure she'd be able to have a child of her own. After two kidney transplants, doctors gave the Omaha woman a laundry list of potential pregnancy complications. Kudrna and husband Adam weighed their options and, in September 2018, welcomed a baby boy.

Joe Nolan couldn't take his son James' pain away. But he could find a way to share it. Nolan got a tattoo that arched across his head, just like his son's scar. James was born with a handful of ailments, including one that regularly requires his skull to be reshaped.

More:
Once winded walking up stairs, Omaha man is back in gym thanks to new cystic fibrosis therapy - Omaha World-Herald

‘We have hope’: Flames assistant GM Chris Snow diagnosed with ALS – National Post

Calgary Flames assistant general manager Chris Snow has been diagnosed with ALS, also known as Lou Gehrigs disease.

The club made that heart-wrenching announcement Wednesday by sharing a letter from his wife, Kelsie.

Snow, an analytics expert, has been on staff at the Saddledome since 2011. The 38-year-old father of two is now enrolled in a clinical trial to treat the disease.

A letter from Kelsie Snow

Dear Hockey Family,

Im here to share news we prayed so hard wed never have.

Chris has been diagnosed with ALS.

ALS is a horrible disease, and when we went to Miami to see one of the best ALS doctors and researchers out there, we prayed hard. We believe there are miracles in the world and maybe, maybe we would get one.

And we did, just not the one wed hoped for.

In the same breath as the doctor telling us that Chris was in the early stages of ALS, he also gave us hope. The next step, he said, was to enroll Chris in a clinical trial for the most encouraging ALS gene therapy treatment to come along. The drug targets a specific genetic mutation that has devastated Chris family. Just over one year ago Chris dad passed away from ALS. We have also lost both of Chris paternal uncles and his 28-year-old cousin to this disease.

In the simplest terms, this drug works by silencing the effects of the mutated gene, and in Miami we could see how hopeful the doctor felt. So hopeful that when I asked if he believes this drug could stop progression entirely, he said, We dont know, but its not outside the realm of possibility.

And so we are leaning into that possibility, as hard as we can, working to stay positive and living with intention every single day. And now we believe we have results to lean into as well.

Chris has been in the trial for several months, and while we do not know whether he is on the actual drug or a placebo, the disease since that initial dose does not seem to have progressed. His right hand and forearm remain the only affected areas of his otherwise strong and healthy body.

Someone has to be the first person to live with ALS rather than die from it, and one thing Ive always known about Chris is that he finds a way. No matter the obstacle, no matter how unprecedented the situation may be he always, always finds a way.

We know that our hockey family will want to help, and we appreciate that so much. Heres how you can do that.

Be positive and hopeful with us, pray for us in whatever way you pray, and dont treat us most especially Chris differently than you always have. He is the same person today he was yesterday and he will be the same person tomorrow and in two months and beyond. Hug your family, wring all the joy from each moment of your life, play with your children, and be present all things at which Chris has always been wonderful.

Continue to love us and love our kids. Of all the devastation this diagnosis brought, the idea of telling the two of them they were going to lose their dad was the most crushing. But now we have hope and, we believe, a different story.

The most tangible way you can help us is by donating to research. ALS is a rare disease, and rare diseases arent easily cured. Fewer than 20,000 people in North America are estimated to be living with ALS. Of them only 2,000 are living with familial ALS, the kind caused by a gene passed down within a family. And yet this has torn through Chris family. At the University of Miami researchers are focused on developing treatments for ALS, including those with familial forms of the disease. They have pioneered the study of the pre-symptomatic stage of ALS with the goal of one day being able to delay or even prevent the disease. With your help they can afford to do more and do it faster. To donate, please visit this link:http://uom.convio.net/goto/chrissnow

The next most powerful thing you can do for us is to believe in this treatment. Our hope is rooted in the results we believe we are seeing and in the optimism expressed by doctors who have spent their careers studying this disease.

As our neurologist said after Chris received his first dose in the trial Were here to make history.

Kelsie Snow

Read this article:
'We have hope': Flames assistant GM Chris Snow diagnosed with ALS - National Post

Augmented Reality is the Future of Healthcare Industry – AiThority

Augmented Reality is already disrupting Healthcare, changing the way physicians see data and their patients. Its being used for patient and doctor education, surgical visualization, disease simulation, and may even help save lives enhancing patient treatments, and outcomes.

Its even making surgery safer, providing surgeons with 3D overlays to enhance the visibility of organs, veins, and diagnosis reports, cutting down on the chance for error. In addition, Augmented Reality plays a crucial role in education, providing real-life simulation surgeries for doctors without the risk of getting it wrong with a live patient. Neurosurgeons have even used Augmented Reality to remove a three-centimeter wide brain tumor in a patients parietal lobe. The best part all of this is being done by ARs ability to blend digital imagery with the real world.

By 2023, the global Augmented reality in Healthcare could become a $1.32 billion market. All of that is creating a sizable opportunity for companies including NexTech AR Solutions, Intuitive Surgical Inc., Medtronic PLC, Abiomed Inc., and Thermo Fisher Scientific Inc.

Read More: Measuring CCPA Preparedness of Big Data Companies: Facts and Insights

NexTech AR Solutions BREAKING NEWS: NexTech AR Solutions, the leader in Augmented Reality (AR) for e-commerce and AR learning applications is pleased to announce that Sterilis Solutions, one ofthe United Statesmost innovative medical waste device manufacturers, has signed on for both NexTechs WebAR offering as well as its AR University. Todays announcement marks the beginning of a significant new use case and opportunity for NexTechs AR technology in the medical device market, which according to Fortune Business Insights is worth$150 billion. Sterilis will be providing Nextechs AR learning assets to its leading dealers and their Sales teams.

Andy Marshall, CEO, Sterilis stresses the critical importance of using AR technology from NexTech AR Solution, AR is more powerful than a brochure especially with the interaction. He continued, We will use it to educate our customers on the core functions of our device as well as supporting our Sales team in showing off this 1,100 lb. machine to our global dealer network.

Echoing the business impact that NexTech AR can have on leading medical device manufacturers such as Sterilis,Evan Gappelberg, CEO of NexTech stated,

We are excited that Sterilis has adopted NexTechs powerful and industry-leading Augmented Reality offering to drive both its product marketing and client education activities. Because our proprietary 3D Augmented Reality (3D/AR) solution and our AR learning tools works on any AR-enabled mobile phone it will help Sterilis to vastly improve its product demonstration capabilities while significantly enhancing its ability to communicate key competitive features of its products at trade shows and really everywhere. He continues, Sterilis has chosen NexTech as a key business growth partner.

Evan added, Unlike other AR/VR companies which charge a fortune and which often require special software and training, NexTechs easy to use and monthly revenue-focused product suite allows its clients to do more and to sell more. All of this results in a stronger bottom line for our customers and recurring revenues for us.

Read More: Like A Machine: How RPA, ML And AI Deliver Smoother And More Streamlined Processes To Accounts Receivable Departments

Intuitive Surgical Inc.announcedU.S. Food and Drug Administrationclearanceof two innovative technologies for two of the companys da Vincisurgical systems to help improve procedures that require sealing. Intuitives E-100 generator is its first internally developed robotic generator to power two key instruments on the da Vinci X and Xi systems. Vessel Sealer Extend is the flagship instrument in the da Vinci energy sealing portfolio today, and the new SynchroSeal instrument offers enhanced capabilities in general surgery.

We are pleased to receive FDA clearance, providing surgeons an additional tool for the X and Xi da Vinci platforms, saidBob DeSantis, Intuitives Senior Vice President and General Manager for Instruments and Accessories. We developed SynchroSeal and the E-100 generator to help surgeons quickly seal and transect tissue and vessels, facilitating efficient technique.

Medtronic PLCandChristianaCareannounced a collaboration designed to improve outcomes for ChristianaCare patients inDelawareand the surrounding region. The five-year agreement will focus on developing and deploying value-based healthcare initiatives to help ChristianaCare apply the right medical technologies and therapies to patients who may benefit most, with shared financial accountability between ChristianaCare and Medtronic to improve patient outcomes while reducing the cost of care.

Theres a general consensus that a fee-for-service system is not sustainable, and a value-based system is the way to go, but how becomes a question. We want to take that on, but transformation can only happen through collaboration with others with aligned views and aligned reward mechanisms, said Omar Ishrak, Chairman and Chief Executive Officer of Medtronic.

ChristianaCares shared commitment to developing value-based healthcare initiatives makes them an ideal partner for Medtronic.

Abiomed Inc.announced initiation of theST-Elevation Myocardial Infarction Door-to-Unloading (STEMI DTU) Pivotal Randomized Controlled Trial (RCT), which will explore whether unloading the hearts left ventricle for 30 minutes with an Impella heart pumpprior to opening blocked arteries will reduce infarct size after a heart attack and lead to a reduction in future heart failure rates.

The first patient in the multi-center trial was enrolled atSpectrum HealthinGrand Rapids,Michigan, by Kevin Wolschleger, MD. The Impella CP heart pump will be used in the STEMI DTU randomized controlled trial, which will study unloading prior to reperfusion as a therapy to reduce heart failure risk.

We are honored to be the first to enroll in this important study which builds on earlier research that shows promise for slowing the growing epidemic of heart failure and improving outcomes for heart attack patients around the world, said Dr. Wolschleger, an interventional Cardiologist at Spectrum Health. Spectrum Health has one of the longest Impella experiences in the United States, and we are proud and excited to be a part of this pivotal clinical trial that has the promise to bring this paradigm-changing therapy to patients, said David Wohns, MD, Chief of Cardiology at Spectrum Health.

Thermo Fisher Scientific Inc. officially opened its new$90 million viral vector CDMO (contract development and manufacturing organization) site in Lexington, Mass. The 50,000-square-foot facility will add more than 200 jobs and support the development, testing and manufacture of viral vectors, which are critical to advancing new life-saving gene and cell therapies globally. Thermo Fishersinvestment in both the regional economic development of the Commonwealth and its commitment to furthering STEM education for young women will well serveMassachusettsand its communities, said GovernorCharlie Baker. We look forward to working together to continue to build upon and strengthen the states role as a global leader in innovation.

Earlier this year, Thermo Fisher completed the acquisition of Brammer Bio, adding this leading viral vector CDMO to its Pharma Services business. The new Lexington site provides much-needed capacity for viral vector development and manufacturing, which to date has been a bottleneck for Biotech companies. This investment is part of a larger strategy to accelerate commercialization of new therapies by providing a range of services from drug development through clinical trials to commercial manufacturing to support Pharma and Biotech companies of all sizes.

The demand for new gene therapies has outpaced capacity, and were in a unique position to partner with our customers to help them accelerate development and production of medicines that will ultimately benefit patients suffering from rare diseases, said Michel Lagarde, executive Vice President of Thermo Fisher Scientific. Were also excited to create 200 new jobs that support the thriving Biotech industry in Massachusetts.

Read More: Worlds Largest Blockchain Application Competition Klaytn Horizon Winners Announced

Read more from the original source:
Augmented Reality is the Future of Healthcare Industry - AiThority

The Best Augmented Reality Stocks for 2020 – PR Newswire UK

Financialnewsmedia.com News Commentary

PALM BEACH, Florida, Dec. 17, 2019 /PRNewswire/ -- Augmented reality is already disrupting healthcare, changing the way physicians see data and their patients. It's being used for patient and doctor education, surgical visualization, disease simulation, and may even help save lives enhancing patient treatments, and outcomes, according to Med City News. It's even making surgery safer, providing surgeons with 3D overlays to enhance visibility of organs, veins, and diagnosis reports, cutting down on the chance for error. In addition, augmented reality plays a crucial role in education, providing real-life simulation surgeries for up and coming doctors without the risk of getting it wrong with a live patient. Neurosurgeons have even used augmented reality to remove a three-centimeter wide brain tumor in a patient's parietal lobe. The best part all of this is being done by AR's ability to blend digital imagery with the real world. By 2023, says Market Research Future, the global augmented reality in healthcare could become a $1.32 billion market. All of that is creating sizable opportunity for companies including NexTech AR Solutions(OTCQB: NEXCF)(CSE: NTAR), Intuitive Surgical Inc. (NASDAQ:ISRG), Medtronic PLC (NYSE:MDT), Abiomed Inc. (NASDAQ:ABMD), and Thermo Fisher Scientific Inc. (NYSE:TMO).

NexTech AR Solutions(OTCQB: NEXCF)(CSE: NTAR) BREAKING NEWS: NexTech AR Solutions, the leader in augmented reality (AR) for eCommerce and AR learning applications is pleased to announce that Sterilis Solutions, one of the United States' most innovative medical waste device manufacturers, has signed on for both NexTech's WebAR offering as well as its AR University. Today's announcement marks the beginning of a significant new use case and opportunity for NexTech's AR technology in the medical device market, which according to Fortune Business Insights is worth $150 billion. Sterilis will be providing Nextech's AR learning assets to its leading dealers and their sales teams. Andy Marshall, CEO, Sterilis stresses the critical importance of using AR technology from NexTech AR Solution, "AR is more powerful than a brochure especially with the interaction." He continues that, "We will use it to educate our customers on the core functions of our device as well as supporting our sales team in showing off this 1,100 lb. machine to our global dealer network."

Echoing the business impact that NexTech AR can have on leading medical device manufacturers such as Sterilis, Evan Gappelberg, CEO of NexTech states: "We are excited that Sterilis has adopted NexTech's powerful and industry-leading augmented reality offering to drive both its product marketing and client education activities. Because our proprietary 3D augmented reality (3D/AR) solution and our AR learning tools works on any AR enabled mobile phone it will help Sterilis to vastly improve its product demonstration capabilities while significantly enhancing its ability to communicate key competitive features of its products at trade shows and really everywhere." He continues, "Sterilis has chosen NexTech as a key business growth partner. Unlike other AR/VR companies which charge a fortune and which often require special software and training, NexTech's easy to use and monthly revenue-focused product suite allows its clients to do more and to sell more. All of this results in a stronger bottom line for our customers and recurring revenues for us."

Other related developments from around the markets include:

Intuitive Surgical Inc. (NASDAQ:ISRG) announcedU.S. Food and Drug Administrationclearanceof two innovative technologies for two of the company's da Vincisurgical systems to help improve procedures that require sealing. Intuitive's E-100 generator is its first internally developed robotic generator to power two key instruments on the da VinciXand Xisystems. Vessel Sealer Extend is the flagship instrument in the da Vinci energy sealing portfolio today, and the new SynchroSeal instrument offers enhanced capabilitiesin general surgery. "We are pleased to receiveFDAclearance, providing surgeons an additional tool for the X andXi da Vinciplatforms," saidBob DeSantis, Intuitive's Senior Vice President and General Manager for Instruments and Accessories. "We developed SynchroSeal and the E-100 generator to help surgeons quickly seal and transect tissue and vessels, facilitating efficient technique."

Medtronic PLC (NYSE:MDT) and ChristianaCare announced a collaboration designed to improve outcomes for ChristianaCare patients in Delaware and the surrounding region. The five-year agreement will focus on developing and deploying value-based healthcare initiatives to help ChristianaCare apply the right medical technologies and therapies to patients who may benefit most, with shared financial accountability between ChristianaCare and Medtronic to improve patient outcomes while reducing the cost of care. "There's general consensus that a fee-for-service system is not sustainable, and a value-based system is the way to go, but 'how' becomes a question. We want to take that on, but transformation can only happen through collaboration with others with aligned views and aligned reward mechanisms," said Omar Ishrak, chairman and chief executive officer of Medtronic. "ChristianaCare's shared commitment to developing value-based healthcare initiatives makes them an ideal partner for Medtronic."

Abiomed Inc. (NASDAQ:ABMD) announced initiation of the ST-Elevation Myocardial Infarction Door-to-Unloading (STEMI DTU) Pivotal Randomized Controlled Trial (RCT), which will explore whether unloading the heart's left ventricle for 30 minutes with an Impella heart pumpprior to opening blocked arteries will reduce infarct size after a heart attack and lead to a reduction in future heart failure rates. The first patient in the multi-center trial was enrolled atSpectrum HealthinGrand Rapids, Michigan, byKevin Wolschleger, MD. The Impella CP heart pump will be used in the STEMI DTU randomized controlled trial, which will study unloading prior to reperfusion as a therapy to reduce heart failure risk. "We are honored to be the first to enroll in this important study which builds on earlier research that shows promise for slowing the growing epidemic of heart failure and improving outcomes for heart attack patients around the world." said Dr. Wolschleger, an interventional cardiologist atSpectrum Health. "Spectrum Health has one of the longest Impella experiences inthe United States, and we are proud and excited to be a part of this pivotal clinical trial that has the promise to bring this paradigm changing therapy to patients," saidDavid Wohns, MD, chief of cardiology atSpectrum Health.

Thermo Fisher Scientific Inc. (NYSE:TMO) officially opened its new$90 millionviral vector CDMO(contract development and manufacturing organization) site inLexington, Mass.The 50,000-square-foot facility will add more than 200 jobs and support the development, testing and manufacture of viral vectors, which are critical to advancing new life-saving gene and cell therapies globally."Thermo Fisher'sinvestment in both the regional economic development of the Commonwealth and its commitment to furthering STEM education for young women will well serveMassachusettsand its communities," said GovernorCharlie Baker. "We look forward to working together to continue to build upon and strengthen the state's role as a global leader in innovation."Earlier this year,Thermo Fishercompleted the acquisition ofBrammer Bio, adding this leading viral vector CDMO to its Pharma Services business.The newLexingtonsite provides much-needed capacity for viral vector development and manufacturing, which to date has been a bottleneck for biotech companies. This investment is part of a larger strategy to accelerate commercialization of new therapies by providing a range of services from drug development through clinical trials to commercial manufacturing to support pharma and biotechcompanies of all sizes. "The demand for new gene therapies has outpaced capacity, and we're in a unique position to partner with our customers to help them accelerate development and production of medicines that will ultimately benefit patients suffering from rare diseases," saidMichel Lagarde, executive vice president of Thermo Fisher Scientific. "We're also excited to create 200 new jobs that support the thriving biotech industry inMassachusetts."

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates Financialnewsmedia.com and MarketNewsUpdates.com, is a third- party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities.The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM was compensated thirty five hundred dollars for news coverage of current press releases issued by NexTech AR Solutions, by a non-affiliated third party. FNM HOLDS NO SHARES OF ANY COMPANY NAMED IN THIS RELEASE.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNM undertakes no obligation to update such statements.

Contact Information:email:editor@financialnewsmedia.com+1(561)325-8757

SOURCE Financialnewsmedia.com

Read the original post:
The Best Augmented Reality Stocks for 2020 - PR Newswire UK

The Politics Of HIV – The Political Football Has Been Fumbled – Seeking Alpha

Introduction

In Trump's 2019 State of the Union Address, the topic of eradicating HIV morphed into a campaign promise with HIV poster child Gilead (GILD) getting most of the accolades due to their advances in prevention drugs. Almost 9 months later (last month), the Trump administration reversed course and sued Gilead which means the position of "HIV poster child" is up for grabs again. Since Trump seems to have knocked the ball out of Gilead's hands, it's unclear if they will be able to repair the damage and reclaim their position or if a new company will rise to the occasion and become the newly anointed.

Trump's 2019 SOTU Address

During Trump's 2019 State of the Union Address, he has announced a strategy to stop the spread of HIV by 2030 by concentrating resources on hot spots where half the new infections occur. After the State of the Union details from the Secretary of Health and Human Services (HHS) Alexa M Azar II, who coordinated the plan, said that their goal was to focus on the 48 countries where half the infections occur and to reduce new infections by 75% along with ending the HIV epidemic in America. After the State of the Union, advocates like Dr. Anthony Facui, director of the National Institute of Allergy and Infectious Diseases, suggested an increase in antiretrovirals and prevention medicine at the Conference on Retroviruses and Opportunistic Infections (CROI). This increased emphasis on treatment was positive for HIV drugmakers like Johnson and Johnson (JNJ), Mylan (MYL), and Pfizer (PFE). Treatment and prevention stocks Gilead (GILD), GlaxoSmithKline (GSK), and Merck (MRK) received the biggest boost from the news, but this year, a new player with disruptive technology might be in the works. Which HIV company could be the biggest beneficiary from this Trump Administration and HHS initiative?

Trump's HIV Initiative

The architect of Trump's plan, Alex Azar, wrote an article after the State of the Union detailing how the Trump Administration and the Department of Health and Human Services are going to reach their goals using existing medicine in conjunction with pre-exposure prophylaxis (PrEP) medication. They are targeting resources to "geographic hotspots", which consists of 48 counties and some other areas and which constitute over 50% of new diagnoses in the United States. Their strategy can be summed up into "Diagnose, Treat, Protect, and Respond." Early detection is critical and getting people on the treatment regimen prevents transmission by keeping the viral loads in check. Additionally, people at risk of contracting HIV have new PrEP drugs like DESCOVY and TRUVADA made by GILD. Their response plan is to use CDC data to rapidly detect and treat growing HIV clusters to prevent new infections. If this model is successful they plan to roll it out on an international scale. The goal of the initiative is to "reduce new infections by 75 percent in the next five years and by 90 percent in the next ten years, averting more than 250,000 HIV infections in that span."

Trump's HIV Plan

The Past Year In Review

There have been many advancements in HIV treatment since this year's State of the Union address. A second patient, in addition to the famous decade-old case of Timothy Ray Brown, was cured of HIV. One drug manufacturer came out with a once a month injectable shot. CRISPR gene-editing technology was used to edit a stem cell implant aimed at curing HIV. Last but not least, a salvage therapy had over a 90% responder's rate with virtually no side effects. If history repeats itself, Trump will double down on his plan to defeat HIV, and unveil more details at the next State of the Union address in February 2020. The State of the Union address is well known for its impact on highlighting winners and losers among the presidential administration's initiatives. HIV is an anticipated winner again this year, but Gilead may not participate because tension has been on the rise between Gilead and the Trump administration. If HIV is mentioned at the State of the Union, it's expected the additional nuances of Trump's HIV plan will come to light the following month at the Conference on Retroviruses and Opportunistic Infections (CROI). This article speculates on some of the big HIV industry players.

The Trump administration, through the Department of Justice (DOJ), filed a patent infringement lawsuit on November 6, 2019 against Gilead seeking damages for patent protection. The Department of Health and Human Services had patents related to pre-exposure prophylaxis (PrEP) for HIV prevention. HHS asserted that GILD "willfully and deliberately induced infringement on the HHS patents." Due to the infringement, GILD allegedly profited from research funded by hundreds of millions of taxpayer dollars and reaped billions in revenues from PrEP through the sale of Truvada and Descovy.

Trump is very volatile, and compared to prior presidents, downright aggressive, so there is a chance that he goes on the attack during his State of the Union address against PrEP drug manufacturers that have taken advantage of the American people. Since PrEP was one of two pillars of Trump's plan, it's safe to say that his administration is now looking for another poster child. On the surface, Merck or GlaxoSmithKline would be the logical alternatives, but something possibly bigger is brewing in the HIV space, which is covered later in this article.

The amfAR Institute for HIV Cure Research is focused on a scientific basis for a cure by the end of 2020. They defined a functional cure as something that would end an individual's lifelong need for drug treatment to keep the virus in check. They identified four key challenges needed to develop a cure: pinpoint the precise locations of the latent reservoirs, determine how they are formed, persist and quantify the amount of virus in the reservoirs, and finally, eradicate them from the body. Gilead is a key partner in this collaboration.

There are many types of T-Cells, but only the CD4+ T-Cell can host the virus. The issue with the existing antiretroviral therapy (ART) is that they work in the blood to reduce the circulating virus, not the virus in the tissues. Only 3-5% of a person's CD4+ cells are circulating at any given time; the remainder are typically compartmentalized in a reservoir in organs such as the brain, lymph nodes, gut, and male/female reproductive tract. According to the MDPI Journal Gilead's planned mechanism of eradication is to use GS-9620, a TLR7 agonist, to force latent HIV from the virus's immune cell reservoir. Once released, broadly neutralizing antibody PGT121 would attach to it and clear it from the blood. This methodology is known as a shock and kill tactic and was presented at the 2018 CROI meeting. Tests in monkeys revealed that this approach delayed viral rebound following ART from 21 days in the control to a median rebound time of 112 days. In July, the company had a phase 1 readout that showed the drug was well tolerated with no Grade 3 or Grade 4 drug-related adverse events. Their finding was that "the results support studies in the potential role of Vesatolimod as part of combination regimens aimed at achieving ART-free control of HIV." The ART-free control verbiage, unfortunately, appears to be well short of a cure. This advance coupled with the patent infringement issue doesn't appear to have enough zest to make it on Trump's radar.

Merck (MRK) had a similar plan called "kick and kill" in which is used a cancer drug called Vorinostat as an activating agent to clear the body of its viral reservoir and get the immune system to respond using it innate immunity. In Merck's RIVER study, released in July 2018, 60 men who recently acquired HIV started ART and got their viral loads to undetectable levels and then received the "kick and kill." This consisted of Vorinostat and an anti-HIV vaccine. Results were very disappointing and showed that there was no impact on copies/mL compared to ART alone. It's important to note that the mice study was marginal in comparison to GILD's monkey study with GS-9620.

Merck is also developing a one-year subcutaneous implant. Anthony Fauci, the head of the National Institute of Allergy and Infection Diseases characterized "an implant for 1 year has enormous potential [...] that could be a game-changing advance." The drug is called MK-8591 or islatravir, and inhibits an enzyme that is needed for replication of HIV. Merck conducted a study in 12 uninfected people and measure the blood levels of islatravir 4 weeks after removal. In half of the cohort that received the higher dose, levels were so high that researchers were able to project that the release of the drug would be 12 - 16 months. No safety issues surfaced in the pilot study. The drug is entering phase 2 studies and has only dosed 12 people so the findings

Viiv Healthcare, the Pfizer and GSK owned HIV joint venture, also is testing cabotegravir in a phase 3 study as an intramuscular injection for the prevention of HIV infection. The phase 2 study results showed overall positive safety and tolerability data, where most patients favored the 12-week injection over pills over daily pills. Due to lower than expected exposure found in some patients in the phase 2 data, the phase 3 study will proceed with a lower dose and 6-week injections.

Almost two years ago, the Gilead PrEP news was extraordinary, but the news now pales in comparison to a group of 5 human patients that have not taken ART's for close to 5 years and have yet to experience a viral rebound that will be discussed later in the article.

Bioethics Observatory Institute of Life Sciences

Timothy Ray Brown, known as the "Berlin patient," was the first person ever cured of HIV, which happened over a decade ago. The second patient cured of HIV is known as the "London patient." Both patients had myeloid leukemia and had total body irradiation to wipe out their T-cells before receiving their stem cell transplant. In both cases, the donor had a genetic mutation - which therefore is present in the donated stem cells - known as a CCR5 delta 32 mutation. People that have the CCR5 delta 32 allele do not have CCR5 receptors on their T-Cells and as a result are immune to HIV. The "Berlin patient" inspired Dr. Monique Nijhuis, from the University Medical Center of Utrecht in the Netherlands to develop a protocol to transplant HIV resistant genes with the intention of eradicating HIV from the patient. The European consortium is analyzing the results of 39 patients who have received transplants from CCR5 delta 32 donors. Dr. Carl Dieffenbach, the director of AIDS research for the National Institute of Health also endorsed this approach at this year's CROI conference and indicated that another was to genetically engineer a patient's immune cells through gene therapy to knock out the CCR5 receptor which is part of HIV's pathway into the T-Cell.

Timothy Ray Brown, right, and Dr. Gero Htter, the Berlin cancer doctor who gave Brown a lifesaving transplant that also cured him of HIV. Photo by Robert Hood / Fred Hutch News Service

There have been 3 HIV vaccine trials for efficacy worldwide. In 2004, the VaxGen candidates failed to offer any protection and halted its phase 3 trial in 2004. Later in 2007, the Phambili study was stopped for safety concerns. An ongoing trial of HVTN100 and HVTN702 is part of a consortium that includes the US Military, the Bill and Melinda Gates Foundation, NIAID, HVTN, GlaxoSmithKline (GSK), and Sanofi Aventis (SNY). Even though vaccines have had a bumpy road, Dieffenbach believes that we could give "remission in absence of eradication." This means that a patient's immune system would be trained to block HIV whenever sleeping HIV infected cells awake, which would be accomplished via injection of HIV blocking antibodies. On a different note, Janssen, a division of JNJ, is enrolling up to 3800 participants in eight countries who will get four vaccine injections over the course of a year. This study, termed the Mosaico study, will vaccinate patients with three different HIV strains, hoping to offer benefit in 65% of the study population. Prophylactic treatment is an ultimate goal in fighting HIV, but it has been elusive because the virus evolves so rapidly that it's difficult to target.

Path Toward a Functional HIV Cure

To understand what a "functional cure" means, it is helpful to recap what exactly HIV is and how existing drugs are currently treating the disease. HIV is a virus that selectively targets a patient's white blood cells known as T-Cells. These T-Cells help fight infections in our body and kill viruses, but the HIV virus exploits a weakness on the surface of the T-Cell. When an HIV virus comes in contact with the CD4 receptor and the CCR5 co-receptor, the virus gains entry into the T-Cell where it can replicate. The current standard of care is called Highly Active Antiretroviral Therapy (HAART) and what it does is target the virus's ability to replicate once already inside the cell, to keep the "viral load" down. It is well understood that keeping the viral load under 50 copies/mL both effectively prevents transmission of the disease and keeps the disease at bay for the existing infectee.

Frontiers in Immunology

Entry/Fusion Inhibitors - Superior Mechanism of Action, But Toxic

The approach to HIV treatment since the advent of HAART has been to mess up the virus' plan to replicate once inside the cell, but in theory, there is a better way. Instead of letting the virus inside the cell, fusion inhibitors block the CCR5 co-receptor and keep the virus outside the cell. With entry/fusion inhibitor treatment the virus can still bind to the CD4 receptor, but it is unable to get close enough to the cell membrane since the CCR5 co-receptor is blocked. Thus, it can't enter the cell and therefore it is unable to replicate. Even more importantly, replication of a virus is what allows for mutation, which ultimately leads to drug resistance as is common with HAART treatment regimens. Historically, fusion inhibitors like Pfizer's maraviroc and Merck's vicriviroc have shown efficacy in keeping viral loads down. However, side effects related to their method of CCR5 inhibition have limited their usage. The effects are apparent as Merck terminated the development of vicriviroc in 2010 and maraviroc has a black box warning for hepatotoxicity.

Corporate Presentation

Next Generation Entry/Fusion Inhibitor - Non Toxic

CytoDyn Inc. (OTCQB:CYDY) has developed a monoclonal antibody CCR5 antagonist called leronlimab to treat HIV with virtually no side effects compared to the nausea, fatigue, and trouble sleeping that come with the HAART regimen. The drug has an impressive safety and efficacy profile. After 24 weeks of leronlimab therapy, 81% of patients had suppressed viral load compared to 43% from the last drug approved in the study's patient population. There were no drug-related Serious Adverse Events (SAEs), giving it one of the best safety profiles in the HIV landscape. Leronlimab also addresses patient compliance as a once-a-week injectable with a 72 hour grace period. The drug also has a fast-track designation and a rolling BLA and is in a position to ask for accelerated approval. It hit its primary endpoint in a pivotal phase 3 trial. They have a very favorable label request with one drug resistance in 3 classes or one drug resistance in 2 classes with limited treatment options. Approval could represent a$1.7 to $3.4 billion market within the first year of approval based on Biovid's Market Research. After expected approval, they plan to do a label expansion to switch to monotherapy maintenance.

Corporate Presentation

It's important to note that 565 leronlimab monotherapy patients have literally thrown away HAART, the standard of care, for close to a year. Five special patients have even been off the side effect heavy HAART 5 years, demonstrating leronlimab's considerable resilience to patients' developing drug resistance, attributable to the fact that leronlimab mostly prevents the virus from ever replicating. Leronlimab may prove to be a superior option over HAART for many patients.

The HAART standard of care, which leronlimab theoretically has the potential to disrupt, represents a >$15 billion franchise for GILD, but the leronlimab monotherapy patients are living ART-free and have complete control of their HIV. Contrast leronlimab's viral rebound data to GILD's GS-9620 animal study, which saw viral loads rebound in 112 days (less than 4 months). Patients in CytoDyn's monotherapy trial are essentially represented by the orange line in the Functional Cure graphic (a few graphics) above, and many patients could remain functionally cured without side effects for years to come, since CytoDyn doesn't have any strong evidence of viral rebounds yet.

Prevention in HIV is all about keeping the viral load under 50 copies/mL, which the FDA considers an undetectable level and incapable of spreading the disease. As a once a week injectable, PRO 140 (leronlimab) offers convenience to the patient, virtually no side effects, and essentially keeps the virus away from the T-Cells and unable to replicate. Here's a nice analogy: HIV is like having little piranha in your blood that only eats T-Cells. The HAART treatment lets the piranha eat the T-Cell but slaughters most but not all of its offspring. Leronlimab, on the other hand, puts a Teflon coating around the T-Cells so the piranha have nothing to eat, so they die from malnutrition.

In a prevention setting, if a person at risk is given leronlimab and exposed to the virus the theory is that the virus will be blocked from replicating and eventually defenses in the body will break it down. This theory might even be applied to patients diagnosed with HIV. If this is done long enough perhaps the virus will eventually break down. Esteban Hernandez-Vargas said that " latently infected resting memory CD4+ T cells are the only cell type in which it has been clearly demonstrated that replication-competent virus can persist for several years in patients." Some patients have been on leronlimab for close to 5 years and at some point, this group of patients would be ideal for that sort analysis to see if they have in fact been cured.

The Thai Red Cross AIDS research center is initiating a PrEP clinical trial in subjects at high risk of HIV infection. Leading scientific advisor Dr. Jonah Sacha is conducting the research and CytoDyn has a Memorandum of Understanding with the Tai Red Cross to develop the HIV PrEP clinical trials. Sacha will also oversee the PrEP cure developments. Supporting PrEP and cure initiatives is the independent leronlimab data from the PRESTIGO Registry Study Group, which is to be presented at CROI. The people in this study are heavily treatment-experienced (HTE) patients that averaged at least 4-classes of drug resistance. The group of patients in this PrEP study are actually less sick than in the leronlimab phase 3 groups, where leronlimab managed to yield a whopping 90% responders rate. The PrEP responders rate to be announced at CROI might be very high. If the majority of HIV positive patients have no side effects, cannot transmit the disease, and has a fully functional immune system, this could be considered a functional cure, allowing Trump to claim a big win.

Throughout the article, little seeds have been planted to accentuate the case for a new player in HIV, who has a disruptive technology, backed by data on long term patients, who possibly could be cured with a drug that has little to no side effects. The CytoDyn story, once again, could be "too good to be true." However, the company is weeks away from a planned BLA submission that will result in a planned drug approval by June 2020, based on its rolling BLA and fast track status. In September, CytoDyn signed a non-binding licensing deal for $90 million from a major distributor. Once the drug is approved, the company has Samsung Biologics ready to provide them with up to $1.0 billion of inventory, based on anticipated drug prices, to meet the expected demand. CytoDyn also signed with distribution partners ready to market the drug. The pieces are in place for CytoDyn to start bringing in billions in revenue next year, except for one key thing. The company needs about $20 to $25 million to fund its operating costs while they are waiting for BLA approval over the next 6 months. This need for either a licensing deal or some type of financing seems to be dampening the enthusiasm for the technology as market participants await closure on financing terms or a licensing deal. It's been eerily quiet in the past two months because no big pharma company has made an even an overture toward what could be the most disruptive technology in the HIV space.

A Gilead investor might consider hedging his bets with the biggest threat in the HIV space. In a worst-case scenario for this investor, nothing happens and CytoDyn doesn't become a political football, then the investor can unwind the hedge after the CROI meeting. If something does happen then the hedge could be wildly profitable and even cover multiple situations included the licensing of the technology by a competitor. In all likelihood, the Trump lawsuit against Gilead could be much ado about nothing, as are most political events. However, can investors ignore 565 patients that were able to throw away their daily HAART regimen and defy big pharma? A 95% responder rate with prolonged remission and favorable safety is good enough to ignite a patient advocacy revolution, and as an investor, you don't want to be caught on the wrong side of the trade.

There are a lot of theories floating out there about how to cure HIV and how to deal with the HIV epidemic. The big picture is that Trump seems stalwart in his approach about eradicating HIV, but therein lies the opportunity. The challenge to investors in the coming months leading up to the State of the Union is figuring out which horse is he going to back. Gilead is the largest HIV drug maker in the world and was the golden child in HIV until the Trump lawsuit hit. This lawsuit should serve notice to GILD investors that they have been targeted by Trump. There is a body of evidence to suggest that CytoDyn has the goods to be crowned the new innovator in HIV. It's definitely a long shot that Trump would elevate a $120 million market cap company as the frontrunner, but it is possible.

The most likely scenario would be Merck and ViiV jockeying for position in their efforts to get top billing and dethrone Gilead. Both of them could potentially negotiate with the administration, but its not clear which company might have the best strategy for PrEP. Whoever is perceived to have the inferior PrEP solution may be forced to seek a license from another PrEP drug maker; however, they would need a drug asset close to approval. Fortunately for CytoDyn, leronlimab approval is only about 6 months away, and if the technology is as robust as presented in this article, a licensee (Merck or ViiV) and CytoDyn could cobble together a viable PrEP program in months and then file for a label expansion.

If Gilead gets wind of this strategy, they might try to play "spoiler", which would be a great outcome for CytoDyn. Regardless, the bottom line is that Trump hit Gilead hard, and they could be on the ropes in the PrEP race. The question for investors is: does Gilead wake up in time to fight back, or does Merck, ViiV, or possibly a CytoDyn collaboration with one of them knock Gilead out?

Disclosure: I am/we are long CYDY. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Editor's Note: This article covers one or more microcap stocks. Please be aware of the risks associated with these stocks.

Read the original here:
The Politics Of HIV - The Political Football Has Been Fumbled - Seeking Alpha

Genentech’s Fixed-dose Subcutaneous Combination of Perjeta and Herceptin Comparable to Intravenous Formulations in People With HER2-positive Breast…

Dec. 12, 2019 06:05 UTC

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new data from the Phase III FeDeriCa study which showed the investigational fixed-dose combination (FDC) of Perjeta (pertuzumab) and Herceptin (trastuzumab) with hyaluronidase, administered by subcutaneous (SC) injection in combination with intravenous (IV) chemotherapy, demonstrated non-inferior levels of Perjeta in the blood (pharmacokinetics) and comparable efficacy and safety to standard IV infusions of Perjeta plus Herceptin and chemotherapy in eligible people with HER2-positive early breast cancer (EBC).

These new data, from a primary analysis of the FeDeriCa study, will be presented in a spotlight session (Abstract #PD4-07) at 7:00 a.m. CST today at the 2019 San Antonio Breast Cancer Symposium (SABCS).

SC administration of the FDC takes approximately eight minutes for the initial loading dose and approximately five minutes for each subsequent maintenance dose. This is compared to approximately 150 minutes for infusion of a loading dose of Perjeta and Herceptin using the standard IV formulations, and between 60-150 minutes for subsequent maintenance infusions of the two medicines.

This fixed-dose subcutaneous combination has the potential to provide a quicker and less invasive method of administration for people with HER2-positive breast cancer being treated with Perjeta and Herceptin, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. This is the first time that we have brought together two of our targeted antibodies as a single subcutaneous injection that can be administered in just minutes.

The FeDeriCa study met its primary endpoint, with SC administration of the FDC showing non-inferior levels of Perjeta in the blood during a given dosing interval (Ctrough) when compared to IV administration of Perjeta. The geometric mean ratio (GMR, a type of average used when assessing pharmacokinetics) for the primary endpoint was 1.22 (90% CI 1.14-1.31) with the lower limit of the 90% CI of the GMR=1.140.80 (the pre-specified non-inferiority margin). A secondary endpoint of non-inferior Ctrough of Herceptin was also met, with blood concentrations for people receiving the FDC non-inferior to those receiving IV Herceptin (GMR=1.33 [90% CI 1.24-1.43]; lower limit of 90% CI of GMR=1.240.80). A non-inferiority endpoint was chosen for the study to ensure that people were receiving sufficient dosing with Perjeta and Herceptin as compared to the established IV doses at the same treatment intervals. In addition, rates of total pathological complete response (pCR), a secondary endpoint, were comparable between the treatment arms, with 59.7% of patients receiving the FDC and 59.5% of patients treated with IV Perjeta and Herceptin achieving a total pCR a difference of 0.15% (95% CI -8.67-8.97).

The safety profile of the FDC in combination with chemotherapy was comparable to that of IV administration of Perjeta plus Herceptin and chemotherapy, and no new safety signals were identified, including no meaningful difference in cardiac toxicity. The most common adverse events in both arms were alopecia, nausea, diarrhea and anemia.

In previous studies, SC administration has been shown to be strongly preferred by the majority of patients compared to IV administration of the same medicine, with the most common reason being that administration required less time in the clinic. In the PHranceSCa study, Genentech is currently investigating patient preference for SC administration of the FDC compared to standard IV administration of Perjeta and Herceptin in people with HER2-positive EBC. Interim results of this Phase II study will be presented at a future medical meeting.

About the FeDeriCa study

FeDeriCa is an international, multicenter, two-arm, randomized, open-label, Phase III study evaluating the pharmacokinetics, efficacy and safety of SC injection of the FDC of Perjeta and Herceptin in combination with chemotherapy, compared with standard IV infusions of Perjeta and Herceptin in combination with chemotherapy in 500 people with HER2-positive EBC who are being treated in the neoadjuvant (before surgery) and adjuvant (after surgery) settings. The primary endpoint of the study is minimum levels of Perjeta in the blood during a given dosing interval (Ctrough). Secondary endpoints include safety; minimum levels of Herceptin in the blood during a given dosing interval (Ctrough); and total pCR, meaning there is no tumor tissue detectable in the tissue removed at the time of surgery. The safety profile of Perjeta and Herceptin FDC was comparable with that of Perjeta and Herceptin administered intravenously.

About HER2-positive breast cancer

Breast cancer is one of the most common cancers among women worldwide. According to the American Cancer Society, approximately 271,000 people in the United States will be diagnosed with breast cancer, and more than 42,000 will die from the disease in 2019. Breast cancer is not one, but many diseases based on the biology of each tumor. In HER2-positive breast cancer, there is excess HER2 protein on the surface of tumor cells. Approximately 15-20% of breast cancers are HER2-positive based on the result of a diagnostic test.

About the FDC of Perjeta and Herceptin

The FDC of Perjeta and Herceptin is a new SC formulation that combines Perjeta and Herceptin with Halozyme Therapeutics Enhanze drug delivery technology.

Trastuzumab in the FDC is the same monoclonal antibody as in IV Herceptin and pertuzumab in the FDC is the same monoclonal antibody as in IV Perjeta. The mechanisms of action of Perjeta and Herceptin are believed to complement each other as both bind to the HER2 receptor, but in different locations. The combination of Perjeta and Herceptin is thought to provide a more comprehensive, dual blockade of the HER signaling pathways.

Halozymes Enhanze drug delivery technology may enable and optimize SC drug delivery for appropriate co-administered therapeutics. The technology is based on a proprietary recombinant human hyaluronidase PH20 (rHuPH20), an enzyme that temporarily degrades hyaluronan a glycosaminoglycan or chain of natural sugars in the body, to aid in the dispersion and absorption of other injected therapeutic drugs.

Current Perjeta and Herceptin IV Indication Statements and Important Safety Information

Perjeta Indication Statements

Perjeta (pertuzumab) is a prescription medicine approved for use in combination with Herceptin (trastuzumab) and chemotherapy for:

Perjeta (pertuzumab) is a prescription medicine approved for use in combination with Herceptin (trastuzumab) and docetaxel in people who have HER2-positive breast cancer that has spread to different parts of the body (metastatic) and who have not received anti-HER2 therapy or chemotherapy for metastatic breast cancer.

Important Safety Information

Side effects with Perjeta

Most serious side effects of Perjeta

Perjeta may cause heart problems, including those without symptoms (such as reduced heart function) and those with symptoms (such as congestive heart failure).

Receiving Perjeta during pregnancy can result in the death of an unborn baby and birth defects.

Other possible serious side effects

Most common side effects

The most common side effects of Perjeta when given with Herceptin and chemotherapy as part of an early breast cancer regimen before surgery are:

Side effects may vary based on chemotherapy regimen.

The most common side effects of Perjeta when given with Herceptin and chemotherapy as part of an early breast cancer regimen after surgery are:

The most common side effects of Perjeta when given with Herceptin and docetaxel for treatment of breast cancer that has spread to other parts of the body (metastatic) are:

Patients are encouraged to report side effects to Genentech and the FDA. Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555.

Patients should talk to a healthcare professional for more information about the benefits and risks of Perjeta.

Please see the Perjeta full Prescribing Information for additional Important Safety Information, including most serious side effects, at http://www.perjeta.com.

Herceptin Indication Statements

Adjuvant Breast Cancer

Herceptin is approved for the treatment of early-stage breast cancer that is Human Epidermal growth factor Receptor 2-positive (HER2+) and has spread into the lymph nodes or is HER2-positive and has not spread into the lymph nodes. If it has not spread into the lymph nodes, the cancer needs to be estrogen receptor/progesterone receptor (ER/PR)-negative or have one high-risk feature.* Herceptin can be used in several different ways:

Patients are selected for therapy based on an FDA-approved test for Herceptin.

*High risk is defined as ER/PR-positive with one of the following features: tumor size greater than 2 cm, age less than 35 years, or tumor grade 2 or 3.

Metastatic Breast Cancer

Herceptin has two approved uses in metastatic breast cancer:

Patients are selected for therapy based on an FDA-approved test for Herceptin.

Important Safety Information

Possible serious side effects with Herceptin

Not all people have serious side effects, but side effects with Herceptin therapy are common.

Although some people may have a life-threatening side effect, most do not.

A patients doctor will stop treatment if any serious side effects occur.

Herceptin is not for everyone. A patient should be sure to contact their doctor if they are experiencing any of the following:

HEART PROBLEMS

These include heart problemssuch as congestive heart failure or reduced heart functionwith or without symptoms. The risk for and seriousness of these heart problems were highest in people who received both Herceptin and a certain type of chemotherapy (anthracycline). In a study of adjuvant (early) breast cancer, one patient died of significantly weakened heart muscle. A patients doctor will check for signs of heart problems before, during, and after treatment with Herceptin.

INFUSION REACTIONS, including:

These signs usually happen within 24 hours after receiving Herceptin.

A patient should be sure to contact their doctor if they:

Are a woman who could become pregnant, or may be pregnant

Herceptin may result in the death of an unborn baby or birth defects. Contraception should be used while receiving Herceptin and for seven months after a patient's last dose of Herceptin. If a patient is or becomes pregnant while receiving Herceptin or within seven months after their last dose of Herceptin, the patient should immediately report Herceptin exposure to Genentech at (888) 835-2555.

Have any signs of SEVERE LUNG PROBLEMS, including:

A patients doctor may check for signs of severe lung problems when he or she examines the patient.

Have LOW WHITE BLOOD CELL COUNTS

Low white blood cell counts can be life threatening. Low white blood cell counts were seen more often in patients receiving Herceptin plus chemotherapy than in patients receiving chemotherapy alone.

A patients doctor may check for signs of low white blood cell counts when he or she examines the patient.

Side effects seen most often with Herceptin

Some patients receiving Herceptin for breast cancer had the following side effects:

A patient should contact their doctor immediately if they have any of the side effects listed above.

Patients are encouraged to report side effects to Genentech and the FDA. Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555.

Patients should talk to a healthcare professional for more information about the benefits and risks of Herceptin.

Please see the Herceptin full Prescribing Information for additional Important Safety Information, including most serious side effects, at http://www.herceptin.com.

About Genentech in breast cancer

Genentech has been advancing breast cancer research for more than 30 years with the goal of helping as many people with the disease as possible. Our medicines, along with companion diagnostic tests, have substantially improved outcomes for HER2-positive breast cancer. As our understanding of breast cancer biology rapidly improves, we are working to identify new biomarkers and approaches to treatment for other subtypes of the disease, including triple-negative and hormone receptor-positive.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191211006028/en/

View post:
Genentech's Fixed-dose Subcutaneous Combination of Perjeta and Herceptin Comparable to Intravenous Formulations in People With HER2-positive Breast...

New drug appears to slow effects of cystic fibrosis, offering hope to long-suffering patients – Pocono Record

By Michele Munz, St. Louis Post-Dispatch

Amy Chastain didnt think she would be able to be a mom. She didnt think her child would be born with the same debilitating disease as her.

She didnt think she would live long enough to see this day, when a drug could change her life and most importantly, her sons.

At age 40, Chastain is pushing the limits. The average life expectancy for someone with cystic fibrosis is 41. Chastain had a serious scare two years ago, when she spent more than a month in the hospital because she got so ill. She needed a feeding tube and oxygen tank. She faced the need for a lung transplant.

A breakthrough drug was approved Oct. 21 for 90% of teen and adult patients with cystic fibrosis, a genetic life-threatening disease that causes thick mucus to build up in the lungs and other organs. The drug Trikafta is the first therapy to show dramatic improvement in lung function for a majority of those with the disease.

Knowing the drug showed promising results in studies, families had been waiting anxiously for months for approval by the federal government, which came faster than expected.

Today marks a tremendous breakthrough and exciting news for people with cystic fibrosis, said Dr. Preston Campbell, president of the Cystic Fibrosis Foundation. This milestone is the result of an extraordinary community coming together against great odds, and we are overjoyed that this will mean more people will have effective treatments for their disease.

Chastain began taking the pill a few weeks after it was approved, and within three days, she said she already felt better. After her morning treatments to clear the mucus that settles in her lungs overnight, she had little to cough up.

She has more energy. Shes able to make her bed, keep her wood floors clean and walk to her car without getting winded and having to take breaks.

I read that it takes effect that quickly, but I didnt believe it, Chastain said. But it is. Its been amazing.

Her son, Kyler, is now 14. Having religiously spent his life doing the breathing treatments that can take two to three hours every day, his lungs are still strong.

At his next appointment with a specialist at St. Louis Childrens Hospital in January, they will begin the process of getting Trikafta for him, too.

While the long-term effects are unknown, the hope is that the drug will help slow the progressive damage of the disease.

Its very exciting to think that he hopefully wont ever get as sick as I am, Chastain said. As a mother, you just cant put in to words what that means to me, that he wont have to go through everything that I have. Hopefully, hell just be able to live a long, healthy life.

But theres more to her story, she says. She might not have become a mom if not for a mistake in her husbands genetic test. Because of the mistake, they falsely believed their child would not be born with cystic fibrosis.

I dont know what happened or why, but Im thankful because, while it took a while to get over my anger, I cant imagine not having Kyler, Chastain said. For whatever reason, he is meant to be here, he is meant to have CF.

Her anger turned to thankfulness, she said, and now its turned to hope.

A verty different time

More than 30,000 people suffer from CF in the United States, with 70,000 affected worldwide according to the Cystic Fibrosis Foundation. In Missouri and Illinois, newborn screening tests show about one in 3,500 babies are born with CF, said Dr. Thomas Ferkol, pediatrics professor at Washington University School of Medicine.

Its considered to be a rare or orphan disease, but its one of the more common inherited diseases, Ferkol said.

In people with CF, mutations in a regulator gene cause a defect in the cells covering surfaces of the body the skin, airways, blood vessels and organs. The cells cant maintain their balance of salt and water, causing mucus in various organs to become thick and sticky.

It leads to damage and ultimately, the destruction of organs, Ferkol said.

In the lungs, mucus clogs the airways and traps germs, leading to infections and other complications. Other problems include decreased sweating, digestive problems, poor growth, diabetes and infertility.

Doctors have treated the disease by addressing the symptoms taking drugs to loosen the mucus, using airway clearing devices several times a day and taking supplements to replace pancreatic enzymes.

Trikafta directly addresses the salt-water imbalance by improving the function of defective proteins. It is the first drug to do so in CF patients with the most common gene mutation 90% have at least one copy of the mutation.

A similar drug, Kalydeco, was approved eight years ago, but it worked in only 8% of patients; and the improvements are not as dramatic.

A study of 403 patients for six months (some taking the drug and some taking a placebo) showed Trifakta normalized chloride levels in sweat, improved lung function by 14% and increased body mass.

A small number of patients participated in the study through Washington University, and though doctors did not know who was receiving the drug or placebo, it was obvious, said pulmonary disease specialist Dr. Daniel Rosenbluth. Other severely ill patients have been able to get early access to the drug.

They feel like they are totally different people, Rosenbluth said. I had a man whose wife kept waking him up at night because he was sleeping soundly and his wife thought he was dead.

Because results were so swift and dramatic, doctors have for nearly a year been telling their patients that a promising drug was coming.

We would tell families, OK, get ready, because we are entering a very different time, Ferkol said.

The Food and Drug Administration reviewed and approved Trifakta in just three months.

Doctors attribute the success to the Cystic Fibrosis Foundation, which over 20 years ago began working with a network of academic centers and organized patients for research studies. This made it quicker and easier for pharmaceutical companies to test drugs in development.

Other rare diseases have been trying to duplicate this model, Rosenbluth said.

The drug, made by Boston-based Vertex Pharmaceuticals, comes with a hefty price tag $311,000 a year. Patients are now wading through the process of seeking coverage through their public or private health insurance.

Keeping up hope

Patients still must continue their daily treatment regimens while taking the new pill, Ferkol stressed. The drug has been studied for only a short time, but the hope is that the drug greatly slows the progression of the disease over ones lifetime.

When Ferkol was a resident doctor in 1985, the life expectancy for a patient with CF was just 23 years old, he said.

This drug is opening up all kinds of possibilities I never dreamed to imagine when I was much younger, Ferkol said. If you can intervene early, before damage has occurred, can that profoundly change the trajectory of the disease? Its going to be a very exciting time.

While its not a cure, it is a drug families and doctors have long been waiting for. Studies are already underway in children ages 6 to 11.

Many families have hoped for a day that we could have something we could do for their children that is going to have the effect that this drug has, Ferkol said.

Hannah Krumrey, 20, of St. Charles, Missouri, is waiting on her insurance to approve her application for the drug; while her older sister, Kayla Krumrey, 21, was able to get the drug early and has already been able to sleep through the night and have more energy to get through the day.

The sisters recently went to Greece, and they hope to be able to travel more together.

Their father died a year ago. Hannah Krumrey wishes she could celebrate the drugs approval with him.

We know how happy he would be. This was like, his dream. This was all he ever wanted, she said. He will still be happy from up there, but I wish he was here to experience it with us.

Hes a blessing

Because Chastains symptoms were not yet severe when she was in her 20s, doctors thought she could safely have a child. But she feared passing on her disease.

A person with CF inherits a faulty gene from both mom and dad. If a person inherits one faulty gene and one normal gene, the person will not have symptoms but is a carrier.

Chastain and her husband decided he would get tested to see if he was a carrier. They were overjoyed when it came back negative, she said.

After two miscarriages, Kyler was born. His newborn screening, which is not always accurate, did not show he had CF.

His bowels were greasy, however, which is a sign of the disease. Chastains concerns grew, and when Kyler was 2 years old, tests confirmed he had CF. Chastain said she was devastated.

I thought I had done everything I was supposed to do to prevent the possibility, she said. I was really mad.

Chastain had watched others in her family suffer worse than she did from the disease, and it frightened her.

I was scared for him. I didnt want that life for him, she said. I didnt know what his disease would be like.

Chastain tried to figure out why her husbands test was negative. She asked to see the results. The hospital, however, said the system showed the couple was a no show the day of the test.

She did not want to identify the hospital.

God couldve stepped in. I dont know, she said. I just know hes a miracle. Hes a blessing. No one is to blame.

Chastain calls her son an outdoor kid. He loves playing basketball, hunting, fishing and riding four-wheelers. At 5 feet, 9 inches tall and 150 pounds, no one would know hes sick.

Chastain says he doesnt talk about stuff, but she can tell he gets worried when shes sick.

After seeing her do better on the new drug, Chastain said Kyler asked her if he would feel different too when he starts taking it.

Hopefully, she told him, it will just keep you feeling the way you do now.

Kyler then asked if she could go hunting with them when she feels better.

The Cystic Fibrosis Foundation is not resting. Just nine days after the new drug was approved, the foundation unveiled its Path to a Cure plan challenging scientists around the world to submit proposals that would accelerate finding a cure and allocating half a billion dollars to fund the research through 2025.

Distributed by Tribune Content Agency, LLC

Read the original here:
New drug appears to slow effects of cystic fibrosis, offering hope to long-suffering patients - Pocono Record

Gene Testing for Antidepressants & Psychotropics: Not There Yet – PsychCentral.com

An increasingly common question I get asked is, Will gene testing help my doctor know which antidepressant to prescribe? Popular tests such as GeneSight suggests that they can shorten your road to recovery and how you, as an individual, will respond to specific antidepressant medications.

Does drug-gene testing, also referred to as pharmacogenomics or pharmacogenetics, work? And if so, does it only work for certain types of medications? Lets find out.

The idea of gene-drug testing is pretty simple. By testing your DNA, companies hope to be able to predict your response (or likely non-response) to specific types of antidepressants. Its also being marketed for a number of other diseases and medications.

Just a year ago, GeneSight had some pretty strong marketing language on its site. The company was strongly suggesting its test could help your doctor choose the best antidepressant for you:

Fortunately, the GeneSight genetic test can provide doctors answers that quickly lead to relief. Pharmacogenomic testing helps empower your doctor with the exact information needed to prescribe you the best medication for you. By examining how your DNA responds to specific medications such as antidepressants, this simple, painless test lets doctors know which medications may not work for you, so you can get back to feeling like yourself again. [] Through pharmacogenomic testing, your doctor can identify the correct medication and create a personalized treatment for you.

In the 2018 announcement of its own antidepressant test, another gene-drug testing company called Color says it now analyzes a number of these genes, starting with two that can impact your response to certain mental health medications like Zoloft, Paxil, and Lexapro. The blog entry cites seven research studies, but none of them have anything to do with antidepressants.

Few genetic researchers feel as positive about the current usefulness of gene-drug testing than companies marketing these tests. The American Psychiatric Associations research council reviewed the evidence last year and found that such genetic testing is not really ready for mass consumption.

Greden et al. (2019) looked at using pharmacogenomics directly to help in depression treatment. Because the researchers didnt find a significant difference (either statistically or clinically) in their primary outcome measure, they instead emphasized the statistical significance they found in two of the 25 secondary outcome measures they examined.

In treatment research, scientists increasingly use a statistic called Number Needed to Treat (NNT) that allows for cross-comparisons of the real-world efficacy of different kinds of treatment. The National Institute for Clinical Excellence (NICE) in the UK recommends that for a treatment to be clinically significant, the NNT should be in the single digits.

According to a critique of the researchers (Goldberg et al., 2019), the Greden study had an NNT of 17 for a response to an antidepressant and an NNT of 19 for remission of a depressive episode. Not exactly powerful numbers. In fact, combined with the non-significance of the primary outcome studied, Greden ironically demonstrated that pharmacogenomics doesnt appear to very good at its primary goal of helping to guide antidepressant treatment.

In short, the science today doesnt support the mainstream use of these tests for antidepressants.

Personalized medicine is the new New Thing marketed by anyone who has access to a DNA lab. The problem is that the marketing of gene-drug testing far overshadows the science. In early 2019, the U.S. Food and Drug Administration updated its guidance on gene-drug testing:

[The] FDA is aware of genetic tests that claim results can be used to help physicians identify which antidepressant medication would have increased effectiveness or side effects compared to other antidepressant medications. However, the relationship between DNA variations and the effectiveness of antidepressant medication has never been established. []

Do not change or stop taking any medicine based on a report from a genetic test you took on your own. []

[And to doctors:] If you are using, or considering using, a genetic test to predict a patients response to specific medications, be aware that for most medications, the relationship between DNA variations and the medications effects has not been established.

Goldberg et al. (2019) said it best:

[Researchers] have noted that commercial [] test manufacturers promote their products with a zeal that is disproportionate to the existing evidence base particularly when marketing to the lay public and clinicians who are likely unfamiliar with the limited statistical power of candidate gene association studies.

Youd be wasting your money by purchasing one of these tests in hopes of getting better results from your antidepressant treatment. The science simply doesnt support use of these tests at this time.

Online health information isnt always accurate on this issue even from trusted sources. For instance, the Mayo Clinic suggest these tests can help, but its unclear whether the anonymous, unlisted author of that article has examined the primary research (as there are no research references listed in the article). Harvard Health Publishing, on the other hand, got it right by noting that the research of gene-drug testing showed no evidence of effectiveness.

Someday, the hope is that pharmacogenetics may meaningfully inform treatment decisions, as it does in oncology. But were not yet there.

References

Goldberg, J.F., Rosenblat, J.D., McIntyre, R.S., Preskorn, S.H., de Leon, J. (2019). Letter to the Editor: Clinical versus statistical significance of pharmacogenomic-guided antidepressant therapy: Whats really being measured and marketed? Journal of Psychiatric Research, 114, 208-209.

Greden, J.F., Parikh, S.V., Rothschild, A.J., et al. (2019). Impact of pharmacogenomics on clinical outcomes in major depressive disorder in the GUIDED trial: a large, patient-and rater-blinded, randomized, controlled study. J. Psychiatr. Res. 111, 5967. https://doi.org/10.1016/j.jpsychires.2019.01.003

Related Articles

Read more:
Gene Testing for Antidepressants & Psychotropics: Not There Yet - PsychCentral.com

Honoring the Legacy of Brian Sorrentino | Innovation – Smithsonian.com

The public called it bubble boy disease. Until recently, any infant born with this rare genetic disordera mutation called severe combined immunodeficiency (SCID-X1), linked to the X chromosomehad little chance of surviving outside a small, sterile environment. Brian Sorrentino, a hematologist and gene therapy researcher at St. Jude Childrens Research Hospital in Memphis, Tennessee, made it his lifes mission to cure this disease.

Sorrentino died at 60 in November 2018, before the groundbreaking results of the first trial were published in The New England Journal of Medicine. As a teenager, Sorrentino had been treated for Hodgkins lymphoma, with heavy doses of radiation. This led to various other ailments later in life, including heart disease and then terminal lung cancer.

He felt like there was a reason that he was saved at 17, says his widow, Suzanne Sorrentino, who also lost her first husband to lung cancer. The work he did to save patients with bubble boy disease was it.

The team thats now carrying on Sorrentinos work at St. Jude won the 2019 Smithsonian magazine American Ingenuity Award in the life sciences category. We spoke with his widow to learn more about the man who started it all. A condensed interview is below.

Can you tell us a bit about your husbands background and what brought him to St. Jude?

Brian was from New York. His dad was a radiologist, and he thought being a doctor would be the greatest profession in the world. After medical school, he worked at the National Institutes of Health with Dr. Arthur Nienhuis. When Dr. Nienhuis came to Memphis to be the head of St. Jude, Brian came with him. That was in 1993. It took Brian some time to adjust to the South. During the years I knew him, we were still working on getting him to say yall instead of you guys.

He was divorced, and the father of two grown children, when I met him just over five years ago. Id never met a scientist before. I told him, I just picture you in a lab coat looking at a microscope all day. He told me that was part of it, but it was a lot more than that.

Was he able to experience the joy of seeing his work on SCID-X1 come to fruition?

Oh, yes. When they got started with the trial, he was so excited. He and [fellow St. Jude researcher Dr.] Ewelina [Mamcarz] were just giddy when they had some children enrolled.

Its one thing to be in your lab and think youve got it. But to go over and see the child and see the parents who are just desperate, it made it real. He said some of the greatest days to him were when he got to leave his lab, his part of St. Jude, and go over where the patients are.

When Brian died, they had treated 10 patients, and theyd come from all over the world. In one of my favorite pictures of him, hes holding one of the children and hes got the biggest smile on his face.

Whats it like to watch the legacy of his work unfold and get celebrated?

Its bittersweet. Brian would say, Recognition is really nice, but its not important. Whats important is the science and saving these children. He didnt live to see the paper about the trial published in the New England Journal of Medicine, but he did know that it had been accepted.

When the paper came out in April, there was so much hubbub about it. The St. Jude PR department was just overwhelmed. They thought it would be big, but not as big as it turned out to be. I think Brian would have been a little embarrassed. St. Jude had a symposium to honor Brian and one of his colleagues in June. It was really lovely, but I just think, Damn it, he should be here!

Beyond scientific research, what were some of Brians other passions?

He played guitar. At a big, nice, fancy St. Jude dinner, with everybody all dressed up, he got up onstage and played Mustang Sally with the band that was performing. He was so nervous. I got so sick of that song. Hed played it a thousand times before the dinner because he didnt want to mess up in front of his colleagues.

He loved the Grateful Dead, which I never understood. I went to four Grateful Dead concerts with him, and told him, You owe me. This music is awful. I like music that has a beginning and an end. He would try to explain how wonderful it was. We buried him in a Jerry Garcia T-shirt.

He also loved his Corvette. He drove it on weekends, and that was his fun car. He had a Volkswagen that he would drive to work that he called his beater. He would get in that beater and drive like he was 85 years old. Hed get in his Corvette and go 100 miles an hour. At the symposium St. Jude had to honor him, everybody who spoke, including the head of the hospital, got up and had some horror story of riding with Brian in his Corvette.

He was just witty and wonderful. And there was a whole new world for me when I met him.

Read the original:
Honoring the Legacy of Brian Sorrentino | Innovation - Smithsonian.com

Genentech Announces New Data on Novel Cd20-cd3 Bispecific Cancer Immunotherapies in People With Difficult-to-Treat Lymphomas – Business Wire

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new data on two investigational CD20-CD3 T-cell engaging bispecific antibodies, mosunetuzumab and CD20-TCB, in people with relapsed or refractory (R/R) B-cell non-Hodgkins lymphoma (NHL). Results from the Phase I/Ib GO29781 study of mosunetuzumab, including data from people previously treated with chimeric antigen receptor (CAR) T-cell therapy, will be presented at the 61st American Society of Hematology (ASH) 2019 Annual Meeting during the Plenary Scientific Session. The Plenary Scientific Session highlights the top six abstracts submitted to the meeting, as determined by the ASH Program Committee. Additionally, results from the Phase I/Ib NP30179 study evaluating CD20-TCB as a combination therapy with Gazyva (obinutuzumab) for people with R/R NHL, will be presented.

Despite recent treatment advancements, slow-growing and aggressive non-Hodgkins lymphomas present increasingly difficult management challenges with each subsequent relapse, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. Were encouraged by these early results, which suggest that our novel bispecific cancer immunotherapies may help people with relapsed or treatment-refractory disease who need more options.

The GO29781 study evaluated mosunetuzumab in patients with R/R NHL, including patients who have relapsed following, or are resistant to, CAR T-cell therapy a patient population with limited treatment options. Results from this dose-escalation study showed encouraging efficacy with an objective response rate (ORR) of 62.7 percent (n=42/67) in slow-growing NHL and 37.1 percent (n=46/124) in aggressive NHL. Additionally, data demonstrated a complete response (CR) rate of 43.3 percent (n=29/67) in slow-growing NHL and 19.4 percent (n=24/124) in aggressive NHL. CRs showed durability, with 82.8 percent (n=24/29) of patients with slow-growing NHL remaining in remission up to 26 months off initial treatment and 70.8 percent (n=17/24) of patients with aggressive NHL, remaining in remission up to 16 months off initial treatment. Of the participants who received prior CAR T-cell therapy, the ORR was 38.9 percent (n=7/18), and 22.2 percent (n=4/18) achieved a CR. Adverse reactions included cytokine release syndrome (CRS) in 28.9 percent of patients with 20.0 percent at Grade 1 and 1.1 percent at Grade 3. Grade 3 neurological adverse events occurred in 3.7 percent of patients.

Results from the Phase I/Ib dose-escalation NP30179 study, evaluating CD20-TCB at doses ranging from 0.6 mg to 16 mg plus Gazyva in people with R/R B-cell NHL, showed an ORR of 54 percent (n=15/28) and a CR rate of 46 percent (n=13/28). This included an ORR and CR of 66.7 percent (n=4/6) in people with follicular lymphoma and an ORR of 50.0 percent (n=11/22) and a CR of 40.9 percent (n=9/22) in aggressive NHL. The most frequently observed adverse event across all treatment doses was CRS, occurring in 67.9 percent of patients (n=19/28), with the majority of events being low grade (Grade 1-2).

Both mosunetuzumab and CD20-TCB continue to be evaluated in a robust clinical development program, investigating the treatments as monotherapies and in combination with other therapies, in people with slow-growing and aggressive forms of NHL.

About Genentechs Investigational Bispecifics

Genentech is currently developing two T-cell engaging bispecific antibodies, mosunetuzumab and CD20-TCB, designed to target CD20 on the surface of B-cells and CD3 on the surface of T-cells. This dual targeting activates and redirects a patients existing T-cells to engage and eliminate target B-cells by releasing cytotoxic proteins into the B-cells. Mosunetuzumab and CD20-TCB differ in their structures, and both are being developed by Genentech as part of our ongoing strategy to explore multiple bispecific formats, to identify those that maximize potential clinical benefits for patients. The clinical development programs for mosunetuzumab and CD20-TCB include ongoing investigations of these molecules as monotherapies and in combination with other medicines, for the treatment of people with CD20-positive B-cell non-Hodgkins lymphomas, including diffuse large B-cell lymphoma and follicular lymphoma.

About the GO29781 study

The GO29781 study [NCT02500407] is a Phase I/Ib, multicenter, open-label, dose-escalation study evaluating the safety and pharmacokinetics of mosunetuzumab in people with relapsed or refractory B-cell non-Hodgkins lymphoma. Outcome measures include best objective response rate by revised International Working Group criteria, maximum tolerated dose, and tolerability.

About the NP30179 study

The NP30179 study [NCT03075696] is a Phase I/Ib, multicenter, open-label, dose-escalation study, evaluating the efficacy, safety, tolerability and pharmacokinetics of CD20-TCB. In this study, CD20-TCB is assessed as a single agent and in combination with Gazyva, following pre-treatment with a one-time, fixed dose of Gazyva, in people with relapsed or refractory B-cell non-Hodgkins lymphoma. Outcome measures include overall response rate, complete response rate per Lugano 2014 criteria, maximum tolerated dose, and tolerability.

About Non-Hodgkins Lymphoma

There are two main types of lymphoma: Hodgkins lymphoma and non-Hodgkins lymphoma (NHL). NHL has two subsets, aggressive and indolent (slow-growing).

NHL represents approximately 85 percent of all lymphomas diagnosed. According to the American Cancer Society, it is expected that nearly 74,000 people will be diagnosed with NHL in the United States in 2019, and nearly 20,000 will die from the disease.

Most cases of NHL start in B-lymphocytes, cells that are part of the bodys immune system and help to defend the body against infections. B-cell lymphoma develops when these cells become cancerous and begin to multiply and collect in the lymph nodes or lymphatic tissues such as the spleen.

Gazyva Indications

Gazyva (obinutuzumab) is a prescription medicine used:

Important Safety Information

The most important safety information patients should know about Gazyva

Patients must tell their doctor right away about any side effect they experience. Gazyva can cause side effects that can become serious or life threatening, including:

Who should not receive Gazyva:

Patients should NOT receive Gazyva if they have had an allergic reaction (e.g., anaphylaxis or serum sickness) to Gazyva. Patients must tell their healthcare provider if they have had an allergic reaction to obinutuzumab or any other ingredients in Gazyva in the past.

Additional possible serious side effects of Gazyva:

Patients must tell their doctor right away about any side effect they experience. Gazyva can cause side effects that may become severe or life threatening, including:

The most common side effects of Gazyva in CLL were infusion reactions, low white blood cell counts, low platelet counts, low red blood cell counts, fever, cough, nausea, and diarrhea.

The safety of Gazyva was evaluated based on 392 patients with relapsed or refractory NHL, including FL (81 percent), small lymphocytic lymphoma (SLL) and marginal zone lymphoma (MZL) (a disease for which Gazyva is not indicated), who did not respond to or progressed within 6 months of treatment with rituximab product or a rituximab product-containing regimen. In patients with follicular lymphoma, the profile of side effects that were seen were consistent with the overall population who had NHL. The most common side effects of Gazyva were infusion reactions, low white blood cell counts, nausea, fatigue, cough, diarrhea, constipation, fever, low platelet counts, vomiting, upper respiratory tract infection, decreased appetite, joint or muscle pain, sinusitis, low red blood cell counts, general weakness, and urinary tract infection.

A randomized, open-label multicenter trial (GALLIUM) evaluated the safety of Gazyva as compared to rituximab product in 1,385 patients with previously untreated follicular lymphoma (86 percent) or marginal zone lymphoma (14 percent).The most common side effects of Gazyva were infusion reactions, low white blood cell count, upper respiratory tract infection, cough, constipation and diarrhea.

Before receiving Gazyva, patients should talk to their doctor about:

Patients should tell their doctor about any side effects.

These are not all of the possible side effects of Gazyva. For more information, patients should ask their doctor or pharmacist.

Gazyva is available by prescription only.

Report side effects to the FDA at (800) FDA-1088, or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555.

Please visit http://www.Gazyva.com for the Gazyva full Prescribing Information, including BOXED WARNINGS, for additional Important Safety Information.

About Genentech in Hematology

For more than 20 years, Genentech has been developing medicines with the goal to redefine treatment in hematology. Today, were investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. For more information visit http://www.gene.com/hematology.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

Read more here:
Genentech Announces New Data on Novel Cd20-cd3 Bispecific Cancer Immunotherapies in People With Difficult-to-Treat Lymphomas - Business Wire

FDA Approves Genentech’s Tecentriq Plus Chemotherapy (Abraxane and Carboplatin) for the Initial Treatment of Metastatic Non-Squamous Non-Small Cell…

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) approved Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane [paclitaxel protein-bound; nab-paclitaxel] and carboplatin) for the initial (first-line) treatment of adults with metastatic non-squamous non-small cell lung cancer (NSCLC) with no EGFR or ALK genomic tumor aberrations.

We are pleased to offer this Tecentriq-based combination as a new treatment option that can provide a clinically meaningful survival benefit for people with non-squamous non-small cell lung cancer, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. Todays approval offers another opportunity to help prolong the lives of people with this type of the disease.

This approval is based on results from the Phase III IMpower130 study, which showed Tecentriq in combination with chemotherapy helped people live significantly longer compared to chemotherapy alone (median overall survival [OS] = 18.6 versus 13.9 months; hazard ratio [HR] = 0.80; 95% CI: 0.640.99; p=0.0384) in the intention-to-treat wild-type (ITT-WT) population. The Tecentriq-based combination also significantly reduced the risk of disease worsening or death (progression-free survival; PFS) compared with chemotherapy alone (median PFS=7.2 versus 6.5 months; HR=0.75; 95% CI: 0.630.91; p=0.0024) in the ITT-WT population.

Safety for the Tecentriq plus chemotherapy combination appeared consistent with the known safety profiles of the individual medicines, and no new safety signals were identified with the combination. Grade 3-4 treatment-related adverse events were reported in 73.2% of people receiving Tecentriq plus chemotherapy compared with 60.3% of people receiving chemotherapy alone.

In lung cancer, Tecentriq is also approved in combination with Avastin (bevacizumab), paclitaxel and carboplatin (chemotherapy), for the initial (first-line) treatment of adults with metastatic non-squamous NSCLC with no EGFR or ALK genomic tumor aberrations. Additionally, Tecentriq is approved by the FDA to treat adults with metastatic NSCLC who have disease progression during or following platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for NSCLC harboring these aberrations prior to receiving Tecentriq. Tecentriq is also approved in combination with carboplatin and etoposide (chemotherapy) for the initial (first-line) treatment of adults with extensive-stage small cell lung cancer (ES-SCLC).

Genentech has an extensive development program for Tecentriq, including nine Phase III studies underway across different types of lung cancer, and multiple ongoing and planned Phase III studies across genitourinary, skin, breast, gastrointestinal, gynecological and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

For those who qualify, Genentech offers patient assistance programs for people taking Tecentriq through Genentech Access Solutions. Doctors can contact Genentech Access Solutions at (866) 422-2377. More information is also available at http://www.Genentech-Access.com.

About the IMpower130 study

IMpower130 is a Phase III, multicenter, open-label, randomized study evaluating the efficacy and safety of Tecentriq in combination nab-paclitaxel and carboplatin versus chemotherapy (nab-paclitaxel and carboplatin) alone for chemotherapy-nave patients with stage IV non-squamous NSCLC. The study enrolled 724 people, of whom 681 were in the ITT-WT population and were randomized (2:1) to receive:

During the treatment-induction phase, people in Arm A received Tecentriq and carboplatin on day 1 of each 21-day cycle, and nab-paclitaxel on days 1, 8 and 15 of each 21-day cycle for 4 or 6 cycles or until loss of clinical benefit, whichever occurred first. People in Arm A received Tecentriq during the maintenance treatment phase until loss of clinical benefit was observed.

During the treatment-induction phase, people in Arm B received carboplatin on day 1 and nab-paclitaxel on days 1, 8 and 15 of each 21-day cycle for 4 or 6 cycles or until disease progression, whichever occurred first. People in Arm B received best supportive care during the maintenance treatment phase. Switch maintenance to pemetrexed was also permitted. People who were consented prior to a protocol revision were given the option to crossover to receive Tecentriq as monotherapy until further disease progression.

The co-primary endpoints were:

About lung cancer

According to the American Cancer Society, it is estimated that more than 228,000 Americans will be diagnosed with lung cancer in 2019, and NSCLC accounts for 80-85% of all lung cancers. It is estimated that approximately 60% of lung cancer diagnoses in the United States are made when the disease is in the advanced stages.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Abraxane is a registered trademark of Abraxis Bioscience, LLC, a wholly owned subsidiary of Celgene Corporation.

Tecentriq U.S. Indications

Tecentriq is a prescription medicine used to treat adults with:

A type of lung cancer called non-small cell lung cancer (NSCLC).

A type of lung cancer called small cell lung cancer (SCLC).

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

What is the most important information about Tecentriq?

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

Tecentriq may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects.

Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Report side effects to Genentech at 1-888-835-2555.

Please visit http://www.Tecentriq.com for the Tecentriq full Prescribing Information for additional Important Safety Information.

About Genentech in personalized cancer immunotherapy

For more than 30 years, Genentech has been developing medicines with the goal to redefine treatment in oncology. Today, were investing more than ever to bring personalized cancer immunotherapy (PCI) to people with cancer. The goal of PCI is to provide each person with a treatment tailored to harness his or her own immune system to fight cancer. Genentech is studying more than 10 cancer immunotherapy medicines across 70 clinical trials alone or in combination with other medicines. In every study we are evaluating biomarkers to identify which people may be appropriate candidates for our medicines. For more information visit http://www.gene.com/cancer-immunotherapy.

About Genentech in lung cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have five approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

See the rest here:
FDA Approves Genentech's Tecentriq Plus Chemotherapy (Abraxane and Carboplatin) for the Initial Treatment of Metastatic Non-Squamous Non-Small Cell...

A #ReUp of 2019: The year when gene therapy, DNA modifications came of age & saved lives – Economic Times

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

6 Nov, 2019

6 Nov, 2019

6 Nov, 2019

6 Nov, 2019

6 Nov, 2019

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

Link:
A #ReUp of 2019: The year when gene therapy, DNA modifications came of age & saved lives - Economic Times

Archives